tag:theconversation.com,2011:/fr/topics/stem-cell-research-943/articlesStem cell research – The Conversation2023-01-11T13:25:40Ztag:theconversation.com,2011:article/1915592023-01-11T13:25:40Z2023-01-11T13:25:40ZTriggering cancer cells to become normal cells – how stem cell therapies can provide new ways to stop tumors from spreading or growing back<figure><img src="https://images.theconversation.com/files/503356/original/file-20230105-19-bvp86r.jpg?ixlib=rb-1.1.0&rect=6%2C6%2C2038%2C2038&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">This image shows pancreatic cancer cells (blue) growing, encased within membranes (red).</span> <span class="attribution"><a class="source" href="https://flic.kr/p/GAACEb">Min Yu/Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC via NIH/Flickr</a>, <a class="license" href="http://creativecommons.org/licenses/by-nc/4.0/">CC BY-NC</a></span></figcaption></figure><p>How cells <a href="https://doi.org/10.3390%2Fijms21186489">become cancerous</a> is a process researchers are still trying to fully understand. Generally, normal cells grow and multiply through controlled cell division, where <a href="https://doi.org/10.3389/fcell.2021.645593">old and damaged cells</a> are replaced after they die by new cells. Sometimes this process stops working, leading cells to start growing uncontrollably and develop into a tumor.</p>
<p>Traditionally, cancer treatments like chemotherapy, immunotherapy, radiation and surgery focus on killing cancer cells. Another type of treatment using stem cells called <a href="https://doi.org/10.1177/1010428317729933">differentiation therapy</a>, however, focuses on persuading cancer cells to become normal cells. </p>
<p><a href="https://scholar.google.com/citations?user=GNSivG8AAAAJ&hl=en">We are</a> <a href="https://chen.uchicago.edu/abhimanyu-thakur-ph-d/">researchers</a> who study how stem cells, or immature cells that can develop into different types of cells, behave in states of health and disease. We believe that stem cells can provide potential treatments for cancer of all types in many different ways.</p>
<h2>How do stem cells contribute to cancer?</h2>
<p><a href="https://www.the-scientist.com/university/brush-up-what-is-stemness-and-pluripotency-70571">Stem cells</a> are unspecialized cells, meaning they can eventually become any one of the various types of cells that make up different parts of the body. They can replenish cells in the skin, bone, blood and other organs during development, and regenerate and repair tissues when they’re damaged.</p>
<p>There are different types of stem cells. Embryonic stem cells are the first cells that initially form after a sperm fertilizes an egg, and can give rise to all other cell types in the human body. Adult stem cells are more mature, meaning they can replace damaged cells only in one type of organ and have a limited ability to multiply. Researchers can <a href="https://doi.org/10.1007%2Fs13238-021-00863-6">reprogram adult stem cells, or differentiated cells</a>, in the lab to act like embryonic stem cells.</p>
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<figcaption><span class="caption">Cells become specialized over the course of development.</span></figcaption>
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<p>Because stem cells can survive longer than regular cells, they have a much higher probability of accumulating genetic mutations that can result in loss of control over their growth and ability to regenerate. This is why many tumors harbor a small subpopulation of cells that <a href="https://doi.org/10.1038%2Flabinvest.2008.14">function like stem cells</a>. These so-called cancer stem cells are <a href="https://doi.org/10.1186/s13578-017-0188-9">thought to be responsible</a> at least in part for cancer initiation, progression, metastasis, recurrence and treatment resistance.</p>
<h2>What is differentiation therapy?</h2>
<p>Accumulating evidence is also showing that cancer stem cells can differentiate into multiple cell types, including noncancerous cells. Researchers are taking advantage of this fact through a type of treatment called <a href="https://doi.org/10.1177/1010428317729933">differentiation therapy</a>. </p>
<p>The concept of differentiation therapy <a href="https://doi.org/10.1038/nrc.2017.103">originated from scientists observing</a> that hormones and cytokines, which are proteins that play a key role in cell communication, can stimulate stem cells to mature and lose their ability to regenerate. It followed that forcing cancer stem cells to differentiate into more mature cells could subsequently stop them from multiplying uncontrollably, making them become normal cells.</p>
<p>Differentiation therapy has been successful in treating <a href="https://doi.org/10.1182/blood-2009-01-198911">acute promyelocytic leukemia</a>, an aggressive blood cancer. In this case, retinoic acid and arsenic are used to block a protein that stops myeloid cells, a type of blood cell derived from the bone marrow, from fully maturing. By allowing these cells to fully mature, they lose their cancerous qualities.</p>
<p>Furthermore, because differentiation therapy doesn’t focus on killing cancer cells and doesn’t surround healthy cells in the body with harmful chemicals, it can be <a href="https://doi.org/10.1182%2Fblood-2009-01-198911">less toxic</a> than traditional treatments.</p>
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<a href="https://images.theconversation.com/files/503362/original/file-20230105-22-8a0umi.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="Microscopy image of acute promyelocytic leukemia" src="https://images.theconversation.com/files/503362/original/file-20230105-22-8a0umi.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/503362/original/file-20230105-22-8a0umi.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=400&fit=crop&dpr=1 600w, https://images.theconversation.com/files/503362/original/file-20230105-22-8a0umi.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=400&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/503362/original/file-20230105-22-8a0umi.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=400&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/503362/original/file-20230105-22-8a0umi.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=503&fit=crop&dpr=1 754w, https://images.theconversation.com/files/503362/original/file-20230105-22-8a0umi.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=503&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/503362/original/file-20230105-22-8a0umi.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=503&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
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<span class="caption">Acute promyelocytic leukemia, as shown in this microscopy image, can be treated with differentiation therapy.</span>
<span class="attribution"><a class="source" href="https://www.gettyimages.com/detail/photo/acute-promyelocytic-leukemia-cells-royalty-free-image/1417347912">jarun011/iStock via Getty Images Plus</a></span>
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<h2>Using stem cells to treat cancer</h2>
<p>There are many other potential ways to use stem cells to treat cancer. For example, cancer stem cells can be <a href="https://doi.org/10.1038/s41392-020-0110-5">directly targeted</a> to stop their growth, or turned into “<a href="https://doi.org/10.1515/iss-2016-0005">Trojan horses</a>” that attack other tumor cells.</p>
<p><a href="https://doi.org/10.1155/2016/1740936">Quiescent cancer stem cells</a>, which don’t divide but are still alive, are another potential drug target. These cells typically play a big role in treatment resistance for various cancer types because they are able to regenerate and avoid death even better than regular cancer stem cells. Their quiescent quality can persist for decades and lead to a cancer relapse. They are also challenging to distinguish from regular cancer stem cells, making them difficult to study.</p>
<p>Researchers can also genetically engineer stem cells to express a protein that binds to a desired target in a cancer cell, increasing the efficacy of treatments by releasing drugs right at the tumor. For example, <a href="https://doi.org/10.3389%2Ffbioe.2020.00043">mesenchymal stem cells</a> derived from bone marrow naturally migrate toward and stick to tumors, and can be used to deliver cancer drugs directly to cancer cells.</p>
<p>Stem cells can also be used to make <a href="https://doi.org/10.1002/wdev.399">organoid models</a>, or miniature versions of organs, to screen potential cancer drugs and study the underlying mechanisms that lead to cancer. </p>
<h2>Challenges in stem cell therapy</h2>
<p>Although, stem cells hold numerous advantages in their use in cancer therapy, they also <a href="https://doi.org/10.18632%2Foncotarget.20798">face various challenges</a>. For example, many current stem cell therapies that aren’t used in combination with other drugs are unable to completely eliminate tumors. There are also concerns about stem cell therapies potentially promoting tumor growth.</p>
<p>Despite these challenges, we believe that stem cell technologies have the potential to open new avenues for cancer therapy. Integrating genetic engineering with stem cells can overcome the major drawbacks of chemotherapeutics, such as toxicity to healthy cells. With further research, cancer stem cell therapies may one day become part of the standard of care for many types of cancer.</p><img src="https://counter.theconversation.com/content/191559/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>The authors do not work for, consult, own shares in or receive funding from any company or organization that would benefit from this article, and have disclosed no relevant affiliations beyond their academic appointment.</span></em></p>Many tumors have cancer stem cells that help them grow and evade treatments. Differentiation therapy forces these cells to mature, stopping growth with less toxicity than traditional treatments.Huanhuan Joyce Chen, Assistant Professor of Molecular Engineering, University of Chicago Pritzker School of Molecular EngineeringAbhimanyu Thakur, Postdoctoral Scholar in Molecular Engineering, University of Chicago Pritzker School of Molecular EngineeringLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/1623052021-06-30T15:16:59Z2021-06-30T15:16:59ZLimits for human embryo research have been changed: this calls for public debate<figure><img src="https://images.theconversation.com/files/407647/original/file-20210622-24-o9o6ri.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Human embryo research allows us to understand normal and abnormal human development. </span> <span class="attribution"><span class="source">Anusorn Nakdee/Shutterstock</span></span></figcaption></figure><p>For 40 years, research into early human development has been guided by the principle that after 14 days, an embryo should not be used for research and must be destroyed. This rule has been part of the law of more than 12 countries. But <a href="https://www.cell.com/stem-cell-reports/fulltext/S2213-6711(21)00263-0">new guidelines</a> released by the <a href="https://www.isscr.org/">International Society for Stem Cell Research</a> have removed this rule. This makes it possible to conduct research on human embryos that are at more advanced stages of development. </p>
<p>Now, countries must revise their laws, policies and guidelines to reflect this change. But first, public debate is crucial to determine the limits of what sort of research should be allowed.</p>
<p>Over the decades <a href="https://pediatrics.aappublications.org/content/108/3/813">human embryo research</a> has allowed us to understand normal and abnormal human development, as well as early genetic diseases and disorders. Studying human embryos, as the earliest forms of human life, can give us insight into why miscarriages occur, and how our complex body systems develop. Human embryos are also important for <a href="https://www.healthline.com/health/stem-cell-research">stem cell research</a>, where researchers try and create cell-based therapies to treat human diseases. </p>
<p>Often, extra embryos are created during <a href="https://www.mayoclinic.org/tests-procedures/in-vitro-fertilization/about/pac-20384716#:%7E:text=In%20vitro%20fertilization%20(IVF)%20is,by%20sperm%20in%20a%20lab.">in vitro fertilisation</a> procedures. These extra embryos may be donated for research. They are cultured (or grown) in a laboratory and can be studied until they reach day 14 post-creation. </p>
<p>The 14-day rule has served as an international standard since 1990 when it was included in the <a href="https://www.legislation.gov.uk/ukpga/1990/37/contents">Human Fertilisation and Embryology Act</a> in the UK. At the time that it was introduced, it was not possible to keep human embryos alive in a laboratory for more than a few days. However, scientists have been recently been able to keep embryos alive for longer periods, between 12 and 13 days. The ethical, legal and social consequences of such research were also important considerations. </p>
<h2>The 14-day rule and the new guidelines</h2>
<p>Although the 14-day rule has been criticised as being arbitrarily decided, there are a number of reasons for the time frame. </p>
<p>After an egg cell is fertilised by a sperm cell, the resulting embryo consists of a few identical cells. Most embryos will implant in the uterus after the 14th day. After this point, the <a href="https://www.sciencedirect.com/topics/biochemistry-genetics-and-molecular-biology/primitive-streak">‘primitive streak’</a> appears, which is the first sign of an embryo’s developing nervous system. The rule also identified the point at which the embryo shows signs of individuation, because it is no longer possible for the embryo to <a href="https://www.vcrmed.com/fertility-treatment/monozygotic-twins/">split into twins</a> after 14 days. Some people reason that due to these events, it is at this stage that a moral being comes into existence, and it would not be ethical to perform research on embryos after this time. </p>
<p>There has been increasing pressure from some researchers to <a href="https://link.springer.com/article/10.1007/s40778-018-0135-7">remove the 14-day rule</a>, or at least <a href="https://www.bmj.com/company/newsroom/extend-14-day-human-embryo-research-limit-to-28-days-urges-ethicist/">extend it,</a> as it prevents critical research from being undertaken. Extending the rule would allow important research into early human development to be done. <a href="https://www.cell.com/stem-cell-reports/fulltext/S2213-6711(21)00263-0">The new guidelines</a> make it possible to do research on embryos older than 14 days if the approval processes of the relevant ethics committees are followed. </p>
<p>A significant problem, however, is that there is no longer any limit on the time frame for research. Would it be permissible to do research on human embryos that are 20 days old or 40 days old? The guidelines specify no limit. The longer a human embryo is allowed to grow, the more recognisably human it becomes. At what point would we regard the research unethical, and at what point does the moral cost outweigh the benefits of research? </p>
<h2>What the law says</h2>
<p>Countries around the world take a <a href="https://www.futuremedicine.com/doi/10.2217/rme-2019-0138">variety of approaches</a> to human embryo research. Some – like Italy and Germany – don’t allow it at all. Others, like the UK, allow research to continue until the embryo is 14 days old, after which it must be destroyed. There are also some which permit embryo research without identifying a limit. Some, like the US, do not have any law regulating it (but there are <a href="http://www.sciencepolicyjournal.org/uploads/5/4/3/4/5434385/wallace_2017.pdf">guidelines</a> which contain reference to the 14-day rule). </p>
<p>In South Africa, reference to the rule is found in the <a href="https://www.up.ac.za/media/shared/12/ZP_Files/health-act.zp122778.pdf">National Health Act (2003)</a>, which states that human embryo research may only be done with permission of the minister, and that the embryos must not be older than 14 days. </p>
<p>International guidelines are not legally binding. But the effect of the revised guidelines is that the international standard for best practice in scientific research has now changed. This means that countries which have implemented the rule in their laws will need to revise them so that they are in line with best practice in science. </p>
<h2>The future of human embryo research</h2>
<p>Human embryo research is a sensitive topic because people are divided on the <a href="https://www.rbmojournal.com/article/S1472-6483(10)61722-1/pdf">moral status of the human embryo</a>. Some people believe that the embryo, as the earliest form of human life, should be protected and not subjected to research at all. Others believe that while an embryo has some moral status, it cannot be protected in the same way as humans are, and may be used for some important research which could ultimately benefit people. </p>
<p>The decision to discard the 14-rule appears to have been made without public input. That does not encourage the public to trust in science, and public engagement should have come before such an an important rule was changed. </p>
<p>There are a number of approaches to working with the revised guidance. Bioethicist Françoise Baylis has suggested that <a href="https://theconversation.com/stem-cell-research-community-drops-14-day-limit-on-human-embryo-research-161616">project-specific time limits should be identified</a>, based on the minimum amount of time required to address the stated research objectives. This would mean that some research would still be subject to the 14-day limit, while other studies would be permitted to exceed it. Another approach would be to keep the 14-day limit as the norm, and consider applications to exceed it <a href="https://www.nature.com/articles/d41586-018-05586-z">case by case</a>. Or the limit could be <a href="https://dx.doi.org/10.15252/emmm.201809437">extended to 28 days</a>. </p>
<p>The coming conversations surrounding embryo research will prove to be very important. The proverbial genie is out of the bottle, and public debate is crucial.</p><img src="https://counter.theconversation.com/content/162305/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Sheetal Soni does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.</span></em></p>The rule, which previously acted as the upper time limit on human embryo research, has been dropped, paving the way for research on older human embryos.Sheetal Soni, Researcher, Lecturer, Attorney, University of KwaZulu-NatalLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/1615782021-05-27T02:32:53Z2021-05-27T02:32:53ZNew global guidelines for stem cell research aim to drive discussions, not lay down the law<figure><img src="https://images.theconversation.com/files/403016/original/file-20210527-23-n1k71z.jpg?ixlib=rb-1.1.0&rect=16%2C0%2C5339%2C3420&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">
</span> <span class="attribution"><span class="source">Paul Sakuma/AP</span></span></figcaption></figure><p>The International Society for Stem Cell Research (ISSCR) today <a href="https://www.isscr.org/policy/guidelines-for-stem-cell-research-and-clinical-translation">released updated guidelines</a> for stem cell research and its translation to medicine. </p>
<p>Developed in response to recent scientific and clinical advances, the revised guidelines provide a series of detailed and practical recommendations that set out global standards for how these emerging technologies should be harnessed.</p>
<p>Stem cell research has <a href="https://www.closerlookatstemcells.org/from-lab-to-you/stem-cells-and-research/">huge potential</a> — it could help pave the way for new therapies for ailments ranging from Parkinson’s disease to childhood kidney failure. But scientific advances in this field can present unique ethical and policy issues beyond that seen in other areas of medical research.</p>
<p>The science is advancing at breakneck pace. Just in the past couple of months, we have seen <a href="https://theconversation.com/researchers-have-grown-human-embryos-from-skin-cells-what-does-that-mean-and-is-it-ethical-157228">model human embryos grown from skin cells</a>, and the creation of <a href="https://theconversation.com/as-scientists-move-closer-to-making-part-human-part-animal-organisms-what-are-the-concerns-159049">human-monkey embryos</a> for use in research. </p>
<p>The ISSCR has long recognised the need to set clear ethical boundaries for stem cell research. Previous guidelines have provided advice on techniques such as the use of human embryos to create stem cells, and set the required standards when using these technologies to create new medicines. </p>
<p>They have also explicitly banned certain practices, such as <a href="https://theconversation.com/dolly-the-sheep-and-the-human-cloning-debate-twenty-years-later-63712">reproductive cloning</a> and the <a href="https://theconversation.com/private-clinics-peddling-of-unproven-stem-cell-treatments-is-unsafe-and-unethical-80608">sale of unproven therapies that claim to be made of stem cells</a>. </p>
<p>The 2021 guidelines — an update on the previous version, released in 2016 — aim to set standards for the many recent advances in stem cell and human embryo research. These include “chimeric” embryos containing cells from humans and other animals, “organoids” grown from stem cells to create tissue that resembles particular human organs, and “models” of human embryos — arrangements of human cells that mimic the early stages of embryo development. </p>
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<p><a href="https://theconversation.com/growing-human-embryos-in-the-lab-and-why-scientists-just-tweaked-the-rules-podcast-161611"><img src="https://images.theconversation.com/files/403160/original/file-20210527-15-1crjmoe.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=212&fit=crop&dpr=1" alt="Promotional image for podcast" width="100%"></a>
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<h2>So what’s new?</h2>
<p>The guidelines contain a clear requirement for certain new stem cell research approaches only to be conducted after a specialised review process. This review should be independent of the researchers, and include community members as well as people with expertise in the relevant science, ethics and law. </p>
<p>This is beyond what is typically required by a university or research institute where medical research is conducted. Besides evaluating the merit of the proposed research, the new reviews should also consider whether there are alternative ways to do the research, the source of stem cells and how they were obtained, and the minimum time required to reach the research goals, particularly in relation to human embryo and related research.</p>
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<img alt="Human embryos" src="https://images.theconversation.com/files/403024/original/file-20210527-13-gn1h3j.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/403024/original/file-20210527-13-gn1h3j.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=422&fit=crop&dpr=1 600w, https://images.theconversation.com/files/403024/original/file-20210527-13-gn1h3j.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=422&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/403024/original/file-20210527-13-gn1h3j.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=422&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/403024/original/file-20210527-13-gn1h3j.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=530&fit=crop&dpr=1 754w, https://images.theconversation.com/files/403024/original/file-20210527-13-gn1h3j.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=530&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/403024/original/file-20210527-13-gn1h3j.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=530&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
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<span class="caption">The new guidelines call for a debate on whether to extend the current 14-day limit for experimentation on human embryos.</span>
<span class="attribution"><span class="source">Oregon Health Sciences/AP</span></span>
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<p>Specialised review is not a new concept. The previous guidelines required it when researchers made stem cells from human embryos or sought to culture human embryos in the lab. But now researchers will now also be required to seek higher review when they create model embryos such as <a href="https://theconversation.com/researchers-have-grown-human-embryos-from-skin-cells-what-does-that-mean-and-is-it-ethical-157228">blastoids</a>, or study the development of animal-human embryos in animal wombs. </p>
<p>Researchers developing <a href="https://theconversation.com/3-parent-ivf-could-prevent-illness-in-many-children-but-its-really-more-like-2-002-parent-ivf-126591">new therapies for mitochondrial disease</a> will also be required to seek higher-level review before attempting to transfer to the uterus of a woman human embryos in which affected mitochondria (a part of the cell’s energy-production apparatus) have been replaced.</p>
<p>Importantly, the revised guidelines also clearly rule out certain activities. These continue to include reproductive cloning and attempts to form a pregnancy in a woman from <a href="https://theconversation.com/chinas-failed-gene-edited-baby-experiment-proves-were-not-ready-for-human-embryo-modification-128454">genetically “edited” human embryos</a> or from model embryos made from stem cells. Prohibited activities also now include using eggs and sperm made from human stem cells for reproduction, or transferring a human-animal chimeric embryo into the uterus of a woman or an ape.</p>
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Read more:
<a href="https://theconversation.com/chinas-failed-gene-edited-baby-experiment-proves-were-not-ready-for-human-embryo-modification-128454">China's failed gene-edited baby experiment proves we're not ready for human embryo modification</a>
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<p>The guidelines also call for a public conversation about whether we should allow limited lab research on human embryos beyond the <a href="https://www.nature.com/articles/d41586-018-05586-z">existing limit of 14 days’ development</a>. Historically, it has not been possible to support human embryonic development outside the body beyond this stage. However, recent advances in human embryo culture raise the possibility that this may now be technically feasible. </p>
<p>Extending the amount of time in culture - in terms of days - could potentially yield new treatments for developmental conditions or infertility, but will also raise concerns about whether possible benefits justify this research. Any decisions to overturn this long-held signpost would need to be carefully deliberated and take into consideration existing law, community values and discussion around what the new limit should be. </p>
<p>The revised guidelines also reinforce the need for informed consent for the collection of human material and participation in stem cell clinical trials, and reiterate that no new stem cell treatment should be made available before it is tested for safety and effectiveness in well-designed and publicly visible clinical trials. The ISSCR continues to condemn the commercial use of unproven stem cell treatments.</p>
<h2>Why do these guidelines matter?</h2>
<p>While stem cell science holds much promise, it is paramount that research is scientifically and ethically rigorous, with appropriate oversight, transparency and public accountability.</p>
<p>The fact these guidelines are driven by experts – including stem cell scientists, doctors, ethicists, lawyers and industry representatives – from across 14 countries indicates a deep sense of responsibility and integrity within the research community, and a desire to ensure science remains in step with community values.</p>
<p>However, these guidelines are recommendations, not laws. </p>
<p>Researchers will need to abide by their respective national or state regulations and ethical standards. Some countries already have regulatory frameworks that are consistent with the new recommendations. In other places there is no national guidance around laboratory and clinical stem cell research at all, or existing law touches on some but not all of the emerging applications of stem cell research. </p>
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Read more:
<a href="https://theconversation.com/as-scientists-move-closer-to-making-part-human-part-animal-organisms-what-are-the-concerns-159049">As scientists move closer to making part human, part animal organisms, what are the concerns?</a>
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<p>For example, in Australia there is already an established pathway for higher-level review of embryo models created from stem cells. However, the same legislation currently bans any attempt to use mitochondrial transfer techniques to create embryos for research or to achieve a pregnancy – both of which are permissible under the new ISSCR guidelines. </p>
<p>Rather than attempting to impose a set of hard-and-fast rules on an ever-evolving research field, the new guidelines attempt to address emerging issues and drive important discussions at domestic level. Ultimately, it is the public and the regulators who will need to set the standards.</p><img src="https://counter.theconversation.com/content/161578/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Megan Munsie has received funding from the Australian Research Council and the Medical Research Future Fund. She is a non-executive director with the National Stem Cell Foundation of Australia, Vice-President of the Australasian Society for Stem Cell Research, member of the MRFF Stem Cell Therapies Mission Expert Advisory Panel and Chair of the Ethics Committee of the International Society for Stem Cell Research (ISSCR) and a member of the taskforce that produced the 2021 ISSCR Guidelines.</span></em></p><p class="fine-print"><em><span>Melissa Little currently receives funding from the NHMRC, MRFF, ARC, PKD USA, PKD Australia. She also has contract research income. She is affiliated with Murdoch Childrens Research Institute and the University of Melbourne. She is the incoming President of the International Society for Stem Cell Research. She also is a Board Member of Research Australia and the Co-Chair of the MRFF Stem Cell Therapies Mission Expert Advisory Panel.</span></em></p>The International Society for Stem Cell Research’s newly released guidelines aim to address new ethical challenges posed by stem cell advances such as model embryos and human-monkey hybrid embryos.Megan Munsie, Head Ethics, Education & Policy in Stem Cell Science and Convener of Stem Cells Australia, The University of MelbourneMelissa Little, Theme Director, Cell Biology, Murdoch Children's Research InstituteLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/923172018-02-27T17:06:36Z2018-02-27T17:06:36ZThe key to treating multiple sclerosis could be inside sufferers’ own bodies<figure><img src="https://images.theconversation.com/files/208110/original/file-20180227-36686-1dyi84f.jpg?ixlib=rb-1.1.0&rect=0%2C29%2C5000%2C3218&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">
</span> <span class="attribution"><a class="source" href="https://www.shutterstock.com/image-vector/nerve-cell-anatomy-detailed-illustration-on-645801253">Tefi/Shutterstock</a></span></figcaption></figure><p>Fat often gets a bad press, but if it didn’t coat the cables that connect our neurons, we’d be in a lot of trouble. Sufferers of multiple sclerosis and a host of other nervous system diseases have first-hand experience of this, with few safe and effective treatment options available. Only now are new treatments appearing on the horizon that might just make a big difference.</p>
<p>In order for us to think, feel and move, information must move around the brain accurately and rapidly. Vital in this process are long wire-like structures called axons, which conduct the electrical currents that encode our thoughts from neuron to neuron.</p>
<p>Most of our axons are sheathed in a fatty substance called <a href="https://www.nationalmssociety.org/What-is-MS/Definition-of-MS/Myelin">myelin</a> which, like the plastic coating on a wire, provides insulation for efficient conduction and protects the axon from damage.</p>
<p>Unfortunately, many diseases damage these myelin sheaths. For example, in <a href="https://theconversation.com/explainer-multiple-sclerosis-32662">multiple sclerosis</a> (MS), the immune system – usually our body’s defence against disease – attacks its own myelin in the brain and spinal cord, leaving the underlying axons exposed. Like a worn-down phone charger, these bare axons can no longer conduct electricity effectively, and are vulnerable to damage. Depending on which cables are damaged, this can cause tingling, weakness, visual problems, and eventually difficulty moving, speaking and swallowing.</p>
<figure> <img src="https://upload.wikimedia.org/wikipedia/commons/4/48/Saltatory_Conduction.gif"><figcaption> An unmyelinated axon and a myelinated axon, side-by-side. Source: www.docjana.com</figcaption></figure>
<p><a href="https://www.mssociety.org.uk/dmts">Most current therapies</a> for MS attempt to stop the immune system from attacking the myelin sheaths. This can reduce damage, but it can’t reverse it. So the condition of many patients deteriorates even while on these drugs. Stem cell transplantation therapy has shown recent promise in treating MS, but such treatments are aggressive and can <a href="https://theconversation.com/can-stem-cell-therapy-really-treat-multiple-sclerosis-63162">seriously endanger patients’ health</a>, requiring chemotherapy to almost completely eliminate the patient’s immune system before attempting to reboot it to an earlier, more healthy stage.</p>
<p>Now, a different kind of stem cell offers exciting potential for a raft of new treatments that could reverse symptoms of MS and other myelin diseases, rather than just slow them – and without the need for transplantation.</p>
<h2>A new hope</h2>
<p>After myelin damage, stem cells called <a href="https://en.wikipedia.org/wiki/Oligodendrocyte_progenitor_cell">OPCs</a> can create specialised brain cells called oligodendrocytes, which send octopus-like arms to wrap new myelin around damaged axons. OPCs are already scattered throughout the brains of MS sufferers, but <a href="https://www.ncbi.nlm.nih.gov/pubmed/23595275">only in some people</a> do they produce enough of the specialised brain cells that regenerate myelin, and therefore <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5006855/">reduce symptoms</a>.</p>
<p>Recent years have seen <a href="https://www.nature.com/articles/nrn.2017.136">great advances</a> in our understanding of how to influence OPC stem cells to respond properly to myelin damage. We can now grow them in hundreds of tiny artificial wells, each containing a different drug and several microscopic axon-mimicking cables, and examine which drugs best kick-start the OPCs into re-myelinating action. <a href="http://www.msdiscovery.org/news/new_findings/12139-novel-remyelination-assay-allows-high-throughput-drug-screening">This innovative lab technique</a> is helping researchers to fast identify the most promising concoctions to take to clinical trials.</p>
<p>Surprisingly, recent discoveries also show that the same immune system responsible for attacking and damaging myelin can also play a beneficial role in regenerating it. For example, immune cells called microglia can gobble up the debris of the old myelin sheaths, clearing the way for new myelin to regenerate. Drugs targeting this process have already <a href="https://www.ncbi.nlm.nih.gov/pubmed/25609628">helped mice to regenerate mylein</a> and will likely be seen in clinical trials soon. What’s more, new <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5006855/">medical imaging technologies</a> will allow us to monitor how well all of these new drugs regenerate myelin inside patients in real time.</p>
<p>The next few years will be an exciting time, as we begin to see clinical data on how these new drugs can help people living with MS. After years of struggle to find an effective treatment, we may just find that the key was inside our bodies all along.</p><img src="https://counter.theconversation.com/content/92317/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Chris McMurran receives funding from MedImmune, and the Jean Shanks Foundation. </span></em></p>All multiple sclerosis sufferers have stem cells with the potential to heal them, but scientists are only just figuring out how to kick them into action.Chris McMurran, MB/PhD Candidate, University of CambridgeLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/671922016-10-17T15:00:43Z2016-10-17T15:00:43ZFirst working eggs made from stem cells points to fertility breakthrough<figure><img src="https://images.theconversation.com/files/142020/original/image-20161017-12425-1q4ia8k.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">
</span> <span class="attribution"><span class="source">Shutterstock</span></span></figcaption></figure><p>Scientists have <a href="http://nature.com/articles/doi:10.1038/nature20104">for the first time</a> shown that fully mature egg cells can be grown in the lab, raising hope for new infertility treatments.</p>
<p>Until now, researchers have only been able to produce cells that resemble sperm or eggs, but which can rarely produce live offspring because of abnormal organisation of their genetic material. But a team at Kyushu University, Japan, have now turned stem cells from mice into mature eggs than can be fertilised and develop into healthy, fertile adults. This could lead to a way for women who can’t naturally produce working eggs to have new ones made from their own cells.</p>
<p><a href="https://stemcells.nih.gov/info/basics/3.htm">Embryonic stem cells</a> are living cells taken from an embryo that have the ability to develop into any other kind of cell. The researchers from Kyushu University team <a href="http://science.sciencemag.org/content/338/6109/971.long">previously demonstrated</a> that, under the right conditions, these cells could be turned into primordial germ cells, immature embryonic versions of sperm and eggs. But because they are immature, these germ cells can’t produce any offspring.</p>
<p>So the researchers <a href="http://nature.com/articles/doi:10.1038/nature20104">adapted their methods</a> to encase the stem cells in other cells taken from a mouse’s foetal gonad (the developing ovary or testis). This recreated an environment more like an ovary and, over a period of four to five weeks, the team saw the stem cells develop into cells resembling mature eggs. </p>
<h2>Fucntioning egg cells</h2>
<p>While the cells looked like mature eggs, the key question was whether they actually were functional egg cells. The team compared their lab-grown eggs with ones from an ovary and found they were the same size and organised their genetic material in similar patterns. </p>
<figure class="align-right ">
<img alt="" src="https://images.theconversation.com/files/142021/original/image-20161017-12463-198jm9y.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/142021/original/image-20161017-12463-198jm9y.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=601&fit=crop&dpr=1 600w, https://images.theconversation.com/files/142021/original/image-20161017-12463-198jm9y.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=601&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/142021/original/image-20161017-12463-198jm9y.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=601&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/142021/original/image-20161017-12463-198jm9y.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=755&fit=crop&dpr=1 754w, https://images.theconversation.com/files/142021/original/image-20161017-12463-198jm9y.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=755&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/142021/original/image-20161017-12463-198jm9y.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=755&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">Mouse embryo.</span>
<span class="attribution"><span class="source">Shutterstock</span></span>
</figcaption>
</figure>
<p>The researchers also showed that their eggs could be fertilised, implanted into a surrogate female and go on to produce live offspring. But only a very small number of their embryos created in this way developed fully to term – just 3.5% of all the embryos they transferred. Importantly though, the team reported that “all the obtained pups grew up normally without evidence of premature death.” </p>
<p>As all good scientists should, the researchers then replicated their experiments to test how robust their technique was. Initially, they used embryonic stem cells in their experiments, but these create an ethical dilemma because an embryo has to be destroyed to produce them.</p>
<p><a href="http://www.sciencedirect.com/science/article/pii/S0092867406009767">In 2006</a>, however, another researcher named Shinya Yamanaka and his team found that turning on just four specific genes in normal adult cells gives them all the potential to develop into other cells just like embryonic stem cells, but without the need to destroy a single embryo. The latest research showed that eggs made from these “induced pluripotent stem cells”, or IPSCs, were just as capable of being fertilised and producing healthy adult offspring as embryonic stem cells.</p>
<h2>Research challenges</h2>
<p>The findings from this study have clear implications for the treatment of human infertility. Being able to manufacture working eggs from regular cells could allow doctors to provide an alternative for women who don’t naturally produce functional eggs. But, as with all research studies like this, there are still some limitations that need to be addressed. </p>
<p>First, the overall success rate of this technique is still low – just 3.5% of all embryos implanted gave rise to live offspring, compared to 30% of those currently used for <a href="http://www.hfea.gov.uk/ivf-success-rate.html">human IVF treatment</a>. Obviously, this would need to be improved, potentially by using different lab conditions, hormonal treatments or by encasing the stem cells in adult gonadal cells rather than foetal ones. However, improving the efficiency of such complex lab techniques can be very difficult.</p>
<p>Second, this study was conducted in mice and not humans. While the two species are similar in the way their eggs and embryos develop, there are <a href="http://dev.biologists.org/content/142/18/3090">some key differences</a>. So scientists still need to prove they can replicate the technique with human cells.</p>
<p>Finally, while the researchers went to great lengths to show that the eggs, embryos and offspring generated in this study were “normal”, the lab-grown eggs did display altered genetic patterns and unusual placenta growth. This means we need to research the full impact of the techniques used in this study on the long-term health of any offspring generated.</p>
<p>Still, the findings from this study open up new possibilities for the preservation and even restoration of fertility in women. As always these kinds of scientific breakthroughs, while there are clear benefits for many people, they also carry potential <a href="https://theconversation.com/worlds-first-genetically-modified-human-embryo-raises-ethical-concerns-40766">ethical implications</a>. But the team at Kyushu University have pushed the boundaries of reproductive biology, opening new avenues that may one day help millions.</p><img src="https://counter.theconversation.com/content/67192/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Adam Watkins does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.</span></em></p>Researchers from Kyushu University, Japan, are the first to turn mice stem cells into mature eggs that can be fertilised.Adam Watkins, Research fellow, Cell & Tissue Biomedicine, Aston UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/542992016-04-13T10:01:43Z2016-04-13T10:01:43ZNew autism research: a nutrient called carnitine might counteract gene mutations linked with ASD risks<figure><img src="https://images.theconversation.com/files/117376/original/image-20160404-27157-4i778m.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Deficiencies in a critical nutrient can lead to an abnormally wired brain. Illustration of a network of nerve cells in the
brain.</span> <span class="attribution"><a class="source" href="https://www.flickr.com/photos/wellcomeimages/14665714320/in/photolist-bxvKyF-9RPXGo-okXBVs-btZc25-oAqCtj-9RMzep-a86z8v-a86XrX-btZcaN-oEdnfk-9RM3Ht-9RMx6R-9RQsjq-9RQsgo-btZc41-9RMyGa-9RMzfp-poJjr3-bGTZvH-a89QnE-btZbT7-4QgsFX-4QguVR-4QkHwA-9RMyLx-pEs81t-9RMxmv-bGTZ66-9RQtYo-a86zcZ-btZbJS-a86S6M-9RQuws-a86RTa-9RPVzh-a86zDa-a86PF2-sksaDS-wmwQUS-wmxgKf-9RQtQU-9RMyDr-a86z6K-9RQtTq-aRkaEX-9RMyJ2">Benedict Campbell, Wellcome Images/Flickr</a>, <a class="license" href="http://creativecommons.org/licenses/by-nc-nd/4.0/">CC BY-NC-ND</a></span></figcaption></figure><p>Autism spectrum disorders (ASDs) affect about <a href="http://dx.doi.org/10.1001/jamapediatrics.2014.210">one percent</a> of the world’s population. In the United States alone, about <a href="http://dx.doi.org/10.1001/jamapediatrics.2014.210">1 in 68 children</a> are on the spectrum, and between <a href="http://dx.doi.org/10.1001/jamapediatrics.2014.210">40 and 60 percent</a> of them are also diagnosed with some degree of intellectual disability.</p>
<p>The annual cost associated with ASD in the United States is high - presently <a href="http://archpedi.jamanetwork.com/article.aspx?articleid=1879723">estimated to be US$236-$262 billion</a>. If diagnoses continue to grow at the current pace, it will exceed <a href="http://dx.doi.org/10.1007/s10803-015-2521-7">$460 billion by 2025</a>, more than the total cost of diabetes.</p>
<p>Scientists still aren’t sure what causes ASD, but evidence suggests it’s probably the result of complex interactions between genetic and environmental factors that affect brain development. So far hundreds of genes whose mutations are associated with ASD have been identified. Many of them are known or predicted to play critical roles in the cells that make up the building blocks of the brain. </p>
<p>Learning more about these genes – and their mutations – might help us understand some of the root causes of ASD, and perhaps find ways to lower the risk that a child will have it.</p>
<p>We decided to take a closer look at mutations in one of these genes, <a href="http://dx.doi.org/10.1016/j.celrep.2016.01.004">called TMLHE</a>, which is required for a critical chemical reaction that lets cells burn fat molecules to produce energy. We wanted to understand how a TMLHE mutation could increase autism risk and whether we could counteract the effect of the mutation. </p>
<h2>Neural stem cells and the developing brain</h2>
<p>When we examined the effect of TMLHE mutations in mice, we found these mutations specifically affect neural stem cells during early stages of brain development.</p>
<figure class="align-left zoomable">
<a href="https://images.theconversation.com/files/117371/original/image-20160404-27136-i11h6d.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/117371/original/image-20160404-27136-i11h6d.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/117371/original/image-20160404-27136-i11h6d.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=615&fit=crop&dpr=1 600w, https://images.theconversation.com/files/117371/original/image-20160404-27136-i11h6d.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=615&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/117371/original/image-20160404-27136-i11h6d.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=615&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/117371/original/image-20160404-27136-i11h6d.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=773&fit=crop&dpr=1 754w, https://images.theconversation.com/files/117371/original/image-20160404-27136-i11h6d.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=773&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/117371/original/image-20160404-27136-i11h6d.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=773&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
<figcaption>
<span class="caption">Stem cells divide in the brain of a zebrafish. A nerve cell (on the outside, turning from purple to white) and another stem cell (on the inside, staying purple), which can itself go on and continue dividing.</span>
<span class="attribution"><a class="source" href="https://www.flickr.com/photos/wellcomeimages/25534509980/in/photolist-9RMz1D-EUp3d5">Paula Alexandre, University College London, Wellcome Images/Flickr</a>, <a class="license" href="http://creativecommons.org/licenses/by-nc-nd/4.0/">CC BY-NC-ND</a></span>
</figcaption>
</figure>
<p>Neural stem cells create all of the specialized cells that make up the brain. When they divide to create two “daughter” cells, one typically becomes a specialized brain cell, such as a neuron, and the other remains a neural stem cell. </p>
<p>This means that the population of neural stem cells is maintained, and the brain building work can continue. Although this process occurs throughout one’s lifetime, it is the most active during embryonic brain development.</p>
<p>If the neural stem cell population is not maintained at the proper level when the brain is developing, there won’t be enough stem cells left to produce the right number and right kind of specialized brain cells. The result is an abnormally wired brain. </p>
<p>We find this to be precisely the problem that TMLHE mutations created in mice. Too often, neural stem cell division created two specialized cells, instead of one specialized cell and one neural stem cell.</p>
<h2>What does a TMLHE mutation do to neural stem cells?</h2>
<p>TMLHE mutations make it difficult for neural stem cells to produce energy, or to maintain a correctly oxidized environment, which is why they often don’t divide properly. </p>
<p>Cells produce energy by processing fat molecules. For this to happen, fat molecules need to get to the mitochondria, the powerhouses of the cell, to be broken down. A nutrient called carnitine helps transport fat to these parts of the cell.</p>
<p>This is where TMLHE comes in. While we can get carnitine from food – milk and meat, for instance – our bodies can also produce it. But the TMLHE gene is required for carnitine synthesis, so a mutation in this gene can lead to carnitine deficiency. This affects energy production in cells and can also result in a cellular environment that is too oxidized for the cell to function properly, which makes problems for the neural stem cell when it divides.</p>
<p>But we also found that this neural stem cell defect is corrected when carnitine is added to TMLHE-deficient cells. This restores their ability to burn fat into energy and to maintain a proper environment within mitochondria, and restores proper cell division behavior to TMLHE-deficient neural stem cells.</p>
<h2>TMLHE mutations are surprisingly common</h2>
<p>Two recent studies have found that the prevalence of TMLHE mutations in human populations may range from about <a href="http://dx.doi.org/10.1073/pnas.1120210109">1 in 350</a> to about <a href="http://dx.doi.org/10.1038/tp.2012.102">1 in 900</a>. In most cases, these people would be unaware that they carry a copy of the defective gene.</p>
<p>Our research raises the possibility that the increased autism risk associated with TMLHE mutations might be effectively managed by making sure the embryo has enough carnitine during critical stages for brain development. It also seems that sufficient carnitine is required at very early stages of pregnancy – far earlier than previously suspected.</p>
<figure class="align-right zoomable">
<a href="https://images.theconversation.com/files/117374/original/image-20160404-27150-11x4yrm.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/117374/original/image-20160404-27150-11x4yrm.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/117374/original/image-20160404-27150-11x4yrm.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=517&fit=crop&dpr=1 600w, https://images.theconversation.com/files/117374/original/image-20160404-27150-11x4yrm.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=517&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/117374/original/image-20160404-27150-11x4yrm.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=517&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/117374/original/image-20160404-27150-11x4yrm.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=650&fit=crop&dpr=1 754w, https://images.theconversation.com/files/117374/original/image-20160404-27150-11x4yrm.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=650&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/117374/original/image-20160404-27150-11x4yrm.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=650&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
<figcaption>
<span class="caption">TLMHE mutations could affect fetal brain development.</span>
<span class="attribution"><a class="source" href="https://upload.wikimedia.org/wikipedia/commons/d/d3/Pregnancy_ultrasound_110322105347_1056300.jpg">Ultrasound via Nevit Dilment via Wikimedia Commons</a>, <a class="license" href="http://creativecommons.org/licenses/by-sa/4.0/">CC BY-SA</a></span>
</figcaption>
</figure>
<p>Either parent can pass on a defective TMLHE gene. Girls have two copies of the gene, inheriting one from each parent. Boys, however, have only one copy of the gene, which they inherit from the mother. If a male fetus inherits the mutant TMLHE gene, it will be unable to produce its own carnitine and will rely on the mother for its carnitine supply. </p>
<p>Hypothetically, a woman who carries a TMLHE mutation could take supplemental dietary carnitine during pregnancy to try to minimize the associated ASD risk – particularly for male babies. </p>
<h2>Carnitine deficiency may be an underestimated ASD risk</h2>
<p>While hundreds of genes are associated with ASD risk, the surprisingly high incidence of TMLHE mutations in human beings suggests the impact of carnitine deficiency on ASD risk may be badly underestimated. This is a particularly interesting possibility given that diet might be a significant contributing factor to ASD risk associated with TMLHE mutations. </p>
<p>Results from <a href="http://dx.doi.org/10.1016/j.celrep.2016.01.004">our mouse study</a>, and a recent study in which an autistic child with a TMLHE mutation was treated with <a href="http://dx.doi.org/10.1002/ajmg.a.37144">carnitine supplementation</a>, suggest that prenatal carnitine supplementation might well be worth considering. However, more research, particularly clinical trials on human populations, will be needed to further establish the role of carnitine in autism prevention.</p><img src="https://counter.theconversation.com/content/54299/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Vytas A. Bankaitis receives funding from The Robert A. Welch Foundation and the National Institutes of Health. </span></em></p><p class="fine-print"><em><span>Zhigang Xie receives funding from National Institute of Health. </span></em></p>A gene mutation that causes problems for neural stem cells – the building blocks of the brain – could be corrected by adding carnitine.Vytas A. Bankaitis, Professor of Chemistry, Texas A&M Health Science Center, Texas A&M UniversityZhigang Xie, Assistant Research Scientist, Texas A&M Health Science Center, Texas A&M UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/458992015-08-24T03:42:42Z2015-08-24T03:42:42ZInformed consent for stem cell research: why it matters and what you should know<figure><img src="https://images.theconversation.com/files/92601/original/image-20150820-7225-1k3yhc0.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">
</span> <span class="attribution"><span class="source">Shutterstock</span></span></figcaption></figure><p><em>This article is part of a series The Conversation Africa is running on stem cell research and therapy. Read the rest of the series <a href="https://theconversation.com/au/topics/african-stem-cells">here.</a></em></p>
<p>Ongoing developments in stem cell science mean that researchers often have no idea how, one year down the line, they will use specimens of human biological material. </p>
<p>But when a scientist takes a swab of your saliva, a sample of your blood or a piece of your skin to research a particular disease, how do you know that it’s going to be used for the intended purpose? And when it is used for research in a different condition, can you take any action? This is where informed consent comes in.</p>
<p>Informed consent is an important part of medical research. It is the process by which a person agrees to be part of a scientific study and then donates their biological material. The person can either agree for their sample to be used in a specific study or give broad consent for it to be used widely for research. </p>
<p>Medical research involving humans and the collection of their biological material should ideally work towards helping the study’s participants to be cured. And when people participate in research, they must be told all the details of the research in a way that leaves them with no uncertainty about their rights in the study. The content should be clearly explained and translated into simple language.</p>
<p>In reality, researchers often give participants information that is not always understood because complicated scientific language is used. This is a major challenge for patients, medical scientists and medical doctors across the world.</p>
<h2>Different stem cells require different consent</h2>
<p>In stem cell research, there are three different types of stem cells that can be donated which need informed consent from participants. </p>
<ul>
<li><p>Adult stem cells. Typically these are found in the person’s skin or grown from a sample of skin cultured in a laboratory from a skin biopsy and can then be used for treatment, such as burns. Until now, most stem cell research has involved obtaining stem cells from a patient for the treatment of their illness or a medical or clinical condition such as a serious burn.</p></li>
<li><p>Multipotent mesenchymal stem cells. These mesenchymal stem cells are taken from bone marrow, the umbilical cord, foetal tissue and fat tissues. Bone marrow cells have been used for treating blood cancers such as leukaemia and blood disorders such as sickle cell disease. </p></li>
<li><p>Pluripotent stem cells. There are two types: those found in a developing embryo (embryonic stem cells) and induced pluripotent stem cells. Embryonic stem cells have become controversial because the process entails terminating the development of a foetus, created by in vitro <a href="http://stemcells.nih.gov/info/basics/pages/basics3.aspx">fertilisation</a> in the laboratory. </p></li>
</ul>
<p>Induced pluripotent stem cells are adult stem cells that are reprogrammed in the laboratory to behave like embryonic stem cells. In the hands of skilled researchers, they can develop into many cell types. Induced pluripotent cells are used as cellular or biological models of certain diseases, called disease-in-the-dish modelling. The results of this type of research could potentially be used to treat some of diseased or damaged tissue.</p>
<p>The ability of pluripotent cells to become a part of a fully functional normal tissue is still unproven. But many animal studies and some human studies are providing <a href="http://stemcell.childrenshospital.org/newsroom/related-topics/tissue-engineering-growing-bladder-cells/">exciting prospects</a>. These stem cells are the most complex.</p>
<p>While they are not yet being used for treatment, they have great potential for future research and the scope and extent of their use is <a href="https://www.uct.ac.za/dailynews/?id=8432">wide</a>. At <a href="http://stemcells.uct.ac.za/">UCT</a> they are currently used as “disease-in-the-dish” models in the laboratory only. It is impossible to anticipate the full range of their future application.</p>
<p>Getting informed consent for induced pluripotent stem cells in the future will need to be done very carefully to assure participants of the ethical use of their specimens. This is because induced pluripotent stem cells can potentially also be used in reproductive cloning. Currently this is banned in South Africa.</p>
<h2>What’s wrong with the forms</h2>
<p>Before any research can go ahead, a patient must complete a customised form. The way researchers get informed consent is guided by recommendations in the <a href="http://www.wma.net/en/30publications/10policies/b3/">Declaration of Helsinki</a>, a set of ethical principles around human experimentation and research using human biological material. It was drawn up in 1964 and has subsequently been revised by the World Medical Association’s committees seven times, including once in South Africa in 1996 and most recently in Brazil in 2013. It is the ultimate guideline around human research ethics. </p>
<p>Researchers are expected to use simple non-scientific terms in the informed consent forms. But because stem cell research is such a complex topic, it may be difficult for patients to understand the scientists’ research intentions. This is particularly true in a developing technology, which is still often in the realm of the <a href="http://www.humangenetics.uct.ac.za/research/neurodegenerative-disorders/inherited-ataxias/">unknown</a>.</p>
<p>In the past, questions have been raised about whether or not patients understand the complicated and scientific content of consent forms. To overcome this, professionally trained staff are being employed to explain the risks and benefits of stem cell research to participants. But this is still a relatively new practice across the globe and South Africa has less than 20 appropriately trained and registered genetic counsellors. There is an <a href="http://www.uct.ac.za/dailynews/?id=8458">urgent need</a> for more.</p>
<p>Their job would be to:</p>
<ul>
<li>help potential participants to navigate through trials; </li>
<li>explain risks, benefits, and therapeutic alternatives; and </li>
<li>provide information about unproven transplants offered outside the bounds of good clinical practice and ethical <a href="http://www.humangenetics.uct.ac.za/teaching/msc-med-genetic-counselling/genetic-counselling-courses-offered-at-uct/">research</a>.</li>
</ul>
<p>For South Africa to continue to develop the capacity to incorporate new bio-medical technologies, such as those in the stem cell field, it must proactively formulate clear guidelines for the oversight of <a href="http://hmpg.co.za/index.php/sajbl/article/view/8408">informed consent</a>. This should ideally cover future anticipated and potential unanticipated use of stem cells in research.</p>
<p><em>This article is based on a paper published in a special South African Medical Research Council Flagship edition of the South African <a href="http://hmpg.co.za/index.php/sajbl/issue/view/497/showToc">Journal</a> of Bioethics and Law.</em></p><img src="https://counter.theconversation.com/content/45899/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Jacquie Greenberg receives funding from the National Research Foundation and SA Medical Research Council for research involving the development of stem cell-based disease-in-the-dish modelling of Inherited Ataxias in Africa.</span></em></p>When it comes to stem cells, the ways that informed consent has been obtained in the past are not sufficient and improvements are needed.Leslie Jacqueline Greenberg, Professor of Human Genetics: Stem Cell Researcher , University of Cape TownLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/459682015-08-24T03:42:35Z2015-08-24T03:42:35ZHow to make sure South Africa’s biobanks balance scientific progress with the law<figure><img src="https://images.theconversation.com/files/92731/original/image-20150821-31386-fv73pv.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Empty sample tubes wait to be filled in a blood and urine sample freezer. South Africa has no legislation governing biobanks that deal with human biological material. </span> <span class="attribution"><span class="source">Reuters/Phil Noble</span></span></figcaption></figure><p><em>This article is part of a series The Conversation Africa is running on stem cell research and therapy. Read the rest of the series <a href="https://theconversation.com/au/topics/african-stem-cells">here</a>.</em></p>
<p>When biobanks were created, the idea was that scientists could have quick access to samples they could use without having to get new specimens every time they needed to do research. </p>
<p>But ongoing developments in science and the commercialisation of scientific based products have heightened the need for legislation and guidelines that could specify both the ethical use and legal implications of samples in biobanks. In South Africa there is no legislation around biobanks and the guidelines are not clear enough. </p>
<p>Biobanks are repositories that store large numbers of human biological materials from donors along with their associated data and the information drawn from this <a href="http://www.scielo.org.za/scielo.php?pid=S0256-95742013000400014&script=sci_arttext&tlng=es">analysis</a>. Biobanks should not be confused with tissue banks, which involve services around cells or tissue from living or dead people for transplants.</p>
<h2>The biobank challenge</h2>
<p>There are two broad categories of biobanks. One category is involved in large cohort studies where tens of thousands of participants are required at a minimum. They are referred to as population biobanks and their function is to promote the <a href="http://www.scielo.org.za/scielo.php?pid=S0256-95742013000400014&script=sci_arttext&tlng=es">health of the population</a>. The second category are <a href="http://researchbriefings.files.parliament.uk/documents/POST-PN-473/POST-PN-473.pdf">disease specific</a>. </p>
<p>In South Africa there are just over a dozen <a href="http://hmpg.co.za/index.php/sajbl/article/view/8406/9680">biobanks</a>, which operate on a smaller scale than their international counterparts. Not all are specific to research, and those involved in human health research are from projects within academic institutions. The country’s National Health Laboratory Services tried to establish a population level biobank but this did not materialise.</p>
<p>Currently there is no legislation governing biobanks.
The <a href="http://www.chr.up.ac.za/undp/domestic/docs/legislation_55.pdf">National Health Act</a> and its regulations, which should be providing direction, is silent on the issue of biobanks.
The National Department of Health’s ethics guidelines make some references to biobanks. It requires all new repositories to have <a href="http://www0.sun.ac.za/research/assets/files/Integrity_and_Ethics/DoH%202015%20Ethics%20in%20Health%20Research%20-%20Principles,%20Processes%20and%20Structures%202nd%20Ed.pdf">approval</a> from the research ethics committee before opening their doors. Ideally this committee should establish the procedures to guide this process and the use of the repository.</p>
<p>But there’s a weakness in this process: oversight from the committees on already established biobanks is not mandatory. </p>
<p>Another problem is that of <a href="http://www.scielo.org.za/scielo.phpid=S0256-95742013000400014&script=sci_arttext&tlng=es">informed consent</a> and individual autonomy around specimens. Informed consent is an ethical and legal doctrine and a necessary component of health research.</p>
<p>In classical research, the consent emphasis lies with the individual who decides how their samples will be used and how long they will be stored for. Because biobank research involves the contributions of large numbers of people, its ethical emphasis generally centres on the <a href="http://sro.library.usyd.edu.au/handle/10765/106615">common good</a>. </p>
<p>Here the challenge comes in with the secondary use of specimens. This is because future research may also involve research questions and methods that were not contemplated at the time of sample collection. Because of this, consent for biobank research cannot be truly “informed”. </p>
<h2>An ideal situation</h2>
<p>At the University of the Witwatersrand, the Human Research Ethics Committee (Medical), which is the oldest ethics committee in the country and among the first to be established in the world, set up a Biobanks Ethics Committee in 2013. This committee reviews applications for the establishment of biobanks and all research around the use of specimens from the approved biobanks. It remains the only biobank specific committee in the country.</p>
<p>For biobanks to operate optimally, their governance should be <a href="http://researchbriefings.files.parliament.uk/documents/POST-PN-473/POST-PN-473.pdf%20Accessed%20on%2016/06/2015">harmonised</a>.</p>
<p>Initial and continuous oversight of biobanks should be provided by research ethics committees. This includes the establishment of a biobank and how it is governed, managed and operated. The biobank must also set up protocols and processes for research activities, consulting with the public and other stakeholders, and establish what the criteria is for sampling and <a href="http://www.oecd.org/sti/biotechnologypolicies/44054609.pdf">selecting participants</a>. </p>
<p><em>This article is based on a paper published in a special South African Medical Research Council Flagship edition of the <a href="http://hmpg.co.za/index.php/sajbl/issue/view/497/showToc">South African Journal of Bioethics and Law</a>.</em></p><img src="https://counter.theconversation.com/content/45968/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>The authors do not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and have disclosed no relevant affiliations beyond their academic appointment.</span></em></p>South Africa has no legislation setting out the rules for biobanks and the guidelines that do exist are not clear or detailed. This leaves the door wide open for unethical practises.Ames Dhai, Director of the Steve Biko Centre for Bioethics and Adjunct Professor and Head of Department of Bioethics at the Faculty of Health Sciences, University of the WitwatersrandSafia Mahomed, Bcom, LLB, LLM, PhD candidate, Steve Biko Centre for Bioethics, Faculty of Health Sciences, University of the WitwatersrandLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/455002015-08-21T04:38:16Z2015-08-21T04:38:16ZSouth Africa’s struggle to control sham stem cell treatments<figure><img src="https://images.theconversation.com/files/92575/original/image-20150820-7225-1sj24wp.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Although safe bone marrow harvesting has taken place in South Africa for more than 50 years, there are many unproven stem cell treatments on offer. </span> <span class="attribution"><span class="source">shutterstock</span></span></figcaption></figure><p><em>This article is part of a series The Conversation Africa is running on stem cell research and therapy. Read the rest of the series <a href="https://theconversation.com/au/topics/african-stem-cells">here</a>.</em></p>
<p>South Africa’s inability to enforce its laws that govern stem cell treatments has resulted in a proliferation of bogus and unproven stem cell treatments being offered to many vulnerable citizens. </p>
<p>Stem cell treatment uses stem cells to repair and replace a patient’s damaged cells or tissues. There are treatments such as bone marrow transplants recognised to be safe and effective to treat blood or immunological disorderslike leukemia, myeloma and aplastic anemia. These have been performed for more than five decades.</p>
<p>But South Africa is one of several countries that has seen a <a href="http://www.futuremedicine.com/doi/abs/10.2217/rme.09.70">sharp increase</a> in the number of patients receiving unproven stem cell-based treatments. The medical community has raised concerns that these widely advertised treatments are potentially harmful, unproven and often fraudulent.</p>
<p>Many of these treatments are still experimental, which means there is no evidence that they are safe. The so-called success of these therapies is anecdotal or is based on self-reports from patients.</p>
<h2>Stem cell tourism destinations</h2>
<p>In the early 2000s South Africa was at the centre of a bogus stem cell treatment scandal, which drew international headlines. Biomark International, also known as Advanced Cell Therapeutics, claimed to be able to cure many diseases including spinal cord injury, multiple sclerosis and amyotropic lateral sclerosis.</p>
<p>More than 800 people allegedly paid exorbitant fees for <a href="http://www.ipscell.com/2012/07/testimony-in-shocking-supermodel-stem-cell-fraud-case-i-lied-to-patients/">largely unproven</a> treatments. The case had legs in the US, the Netherlands and South Africa. One of the alleged perpetrators, Steven van Rooyen, who fled the US and continued his fraudulent activities in South Africa, is still on the list of most wanted fugitives of the US’s Food and Drug Administration’s <a href="http://www.fda.gov/ICECI/CriminalInvestigations/ucm389556.htm">Office of Criminal Investigations</a>.</p>
<p>By 2012, <a href="http://www.ajol.info/index.php/sajbl/article/view/83487/73522">researchers</a> estimated that there were more than 700 clinics offering bogus treatments operating
mostly in developing countries such as Costa Rica, Argentina, China, India, Russia and South Africa.</p>
<p>These clinics had lured patients from industrialised countries, desperate to find treatments for their non-curable diseases. Other popular destinations for <a href="https://theconversation.com/medical-treatment-not-approved-yet-no-problem-welcome-to-circumvention-tourism-35070">stem cell tourism</a> included the Bahamas, Singapore, Mexico, Korea, Thailand, Barbados, Hungary, Malaysia, Japan and Thailand.</p>
<h2>Success stories</h2>
<p>Globally, the failure to contain bogus stem cell providers and enforce legislation is compounded by inaction and some complicity from governments and medical establishments. </p>
<p>The US, Netherlands and <a href="http://www.igz.nl/uk/files/379598">Ireland</a> have, however, been effective in addressing the problem. This is largely because of strict regulations and <a href="http://www.kultur.lu.se/uploads/media/Stem_Cell_Tourism_-_State_of_the_Art_Report.pdf">protective measures</a> which shield vulnerable patients from these operators.</p>
<p>Although most countries have legislation that governs research on human subjects, medical malpractice and licensing laws, some guidelines are not specific to stem cell therapy. While international agreements may help close this regulatory gap, some countries don’t accept or abide by these. </p>
<p>Media reports do, however, play an important part in addressing the problem. For example, a BBC documentary series several years ago revealed a lucrative trade in human fetuses. The fetuses were sourced from the Ukraine and sold to stem cell clinics in the Caribbean. This led to one of the major clinics, the Institute for Regenerative Medicine in <a href="http://web.archive.org/web/20080128173958">Barbados</a> being closed down.</p>
<h2>Local enforcement lacking</h2>
<p>South Africa has laws that govern the regulation and registration of human tissue, medicines, health professions and medical malpractice. </p>
<p>The National Department of Health provides guidelines on ethics in health research. This stipulates that all health research must be approved by a registered and accredited research ethics committee before the study begins.</p>
<p>In addition, the legal requirements to register medicines is governed by the provisions of the Medicines and Related Substances Control Act. Under this act, some types of stem cell therapy can be classified as biological medicine. This means that these products must be registered and undergo safety and quality tests before they can be used. This may include pre-clinical studies in relevant animal models.</p>
<p>But, in some cases, the prescripts of these laws are not exercised by practitioners and there is no enforcement. Stem cells are not registered as biological medicines and the treatments are not subjected to clinical trials or peer review by ethics committees. </p>
<p>Unproven stem cell therapies generally fail to comply with minimal legal, ethical, scientific and medical standards of safety and efficacy, which clinical trials set out to determine.</p>
<p>There is no national watchdog or regulatory body to monitor activities in the field, including patient safety and the ethical standards of healthcare practitioners.</p>
<h2>The medical risks</h2>
<p>As a result, patient safety is compromised. Patients who undergo bogus treatments face several risks. These include: </p>
<ul>
<li><p>not giving informed consent for the stem cell samples, which could then be used unethically without their knowledge;</p></li>
<li><p>expensive therapy despite the accepted norm that experimental treatments should be free;</p></li>
<li><p>no post-treatment care or follow-up monitoring to deal with adverse events;</p></li>
<li><p>absence of patient registries;</p></li>
<li><p>associated medical risks such tumour growth, immunological reactions or unknown long-term health consequences. </p></li>
</ul>
<p>Patients should not be afraid to seek remedy if they are harmed. Registered healthcare providers who provide bogus treatments could face disciplinary action from their respective professional bodies. And disclaimers in contracts signed with bogus providers do not necessarily exonerate these providers from the harm they cause. </p>
<p>If South Africa’s laws and regulations governing the use of human tissue are not amended to close the regulatory gap that has arisen, stem cell tourism will flourish and legitimate stem cell research and its clinical translation will be jeopardised. </p>
<hr>
<p><em>This article based on a paper published in a special South African Medical Research Council Flagship edition of the <a href="http://hmpg.co.za/index.php/sajbl/issue/view/497/showToc">South African Journal of Bioethics and Law</a>.</em></p><img src="https://counter.theconversation.com/content/45500/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Melodie Slabbert works the College of Law, University of South Africa</span></em></p><p class="fine-print"><em><span>Michael Sean Pepper receives research funding from the South African Medical Research Council, The National Research Foundation of South Africa and the National Health Laboratory Services Research Trust.</span></em></p>Unless the South African government tightens its laws that govern stem cell treatments, unproven stem cell therapy will continue to surge.Melodie Labuschaigne, Deputy Executive Dean and Professor of Law, College of Law, University of South AfricaMichael Sean Pepper, Director of the Institute for Cellular and Molecular Medicine, University of PretoriaLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/459272015-08-20T04:32:05Z2015-08-20T04:32:05ZWhy the world needs to keep pace with breakthroughs in stem cell research<figure><img src="https://images.theconversation.com/files/92440/original/image-20150819-10873-w6xl7n.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Demonstrators dressed as embryos gather outside the French parliament to protest laws authorising research on embryonic stem cells. Across the world, countries are implementing additional laws to use treatments that are still in trial phases.
</span> <span class="attribution"><span class="source">Reuters/Benoit Tessier</span></span></figcaption></figure><p><em>This article is part of a series The Conversation Africa is running on stem cell research and therapy. Read the rest of the series <a href="https://theconversation.com/au/topics/african-stem-cells">here</a>.</em></p>
<p>Exploration into the use of stem cells has created a cauldron where scientists and regulators are increasingly pressurised to find ways of fast-tracking promising research into novel therapies.</p>
<p>Many countries are developing guidelines and legislation to balance the provision of stem cell therapies as quickly as possible, while still ensuring the safety and efficacy of treatments.</p>
<p>At the centre of this is the accelerated, or conditional, approval of medicine still under clinical development. This is opposed to medicine already proven to be safe and effective in humans, formally approved by the relevant regulatory authority and legally available for sale to the public. </p>
<p>Although the South African legal systems do not provide for any accelerated or conditional approval, heed should be taken of global developments. This will ensure that legal systems allow local scientific innovation to keep up with global pace setters and that patients are protected. </p>
<h2>Stem cells and investigational medicine</h2>
<p>The use of investigational medicine, as we know it today, came about in the late 1980s. In an attempt to address lengthy drug development cycles and a growing AIDS epidemic, the US was forced to reconsider its strict guidelines around investigational medicine. It eventually <a href="http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.304.9143">amended</a> them to allow access to investigational medicine under certain conditions. </p>
<p>This developed into the Food and Drug Administration’s (FDA) system of expanded access. This allows patients with serious conditions to receive investigational medicine that has not yet undergone formal product approval. </p>
<p>There are three prescribed categories of people who can get expanded access to investigational medicine: </p>
<ul>
<li><p>individuals;</p></li>
<li><p>intermediate-size patient populations; and </p></li>
<li><p>widespread use under a treatment protocol. </p></li>
</ul>
<p>For individual patients, the decision lies with a physician. They need to confirm that the medicine does not pose a greater risk to the patient’s health than the disease itself.</p>
<p>When large numbers of patients are involved, the FDA must still find evidence that it is safe to use. To qualify, the FDA must determine whether the condition is serious or immediately life-threatening, and that there are no alternative satisfactory treatments available. Access to investigational medicine must also not interfere with the necessary clinical trials required to receive formal product approval. </p>
<p>Similar to all other therapeutics that qualify as biological medicine, stem cells must go through lengthy clinical trials to prove their safety and efficacy before they can be approved by the relevant regulatory authority for public use. Before they received the final approval, stem cells remain investigational medicine. </p>
<p>This should not be confused with the countless bogus stem cell treatments often advertised online. Many of these treatments have not:</p>
<ul>
<li><p>undergone clinical trials;</p></li>
<li><p>passed the first phase of these trials; or</p></li>
<li><p>received the go-ahead from authorities to be used as an investigational medicine. </p></li>
</ul>
<h2>Expanded access is gaining momentum</h2>
<p>In many countries, access to stem cell treatments still classified as investigational medicine, is gaining momentum. </p>
<p>In addition to the US’s expanded access guidelines, the <a href="https://www.govtrack.us/congress/bills/114/hr3012/text">Right to Try Act</a> was introduced in July this year. If passed, the bill, which is complementary to similar state laws, will stop the federal government from taking action to stop expanded access of investigational drugs to terminally ill patients. This will severely limit the FDA’s regulatory authority over expanded access in these instances. </p>
<p>Although federal laws trump state laws, twelve states have already enacted so-called <a href="http://www.ncsl.org/documents/health/RighttoTry2015.pdf">“right-to try” legislation</a>. This has created enormous legal ambiguity as these state laws are not really enforceable. But, in practice, the fear of violating the FDA’s requirements causes a reluctance to actually use these state laws. </p>
<p>Similarly, the European Medicines Agency has started a public consultation process of revised guidelines to implement <a href="http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2015/07/WC500190554.pdf">accelerated access</a> and conditional marketing authorisation. These are based on less complete clinical data. This will also accelerate patients’ access to medicines and address unmet medical needs. </p>
<p>The guidelines are specifically aimed at innovative medicines and target diseases where no treatment is available. It could provide patients with a major therapeutic advantage over existing treatments. </p>
<p>This public consultation process is hot on the heels of the highly debated <a href="http://www.eurostemcell.org/story/scientists-raise-alarm-italian-government-rules-unproven-stem-cell-therapy">Stamina Foundation debacle</a>. This involved an Italian court ruling that unproven stem cell therapy on a three-year-old child with <a href="http://www.mayoclinic.org/diseases-conditions/krabbe-disease/basics/definition/con-20029450">Krabbe disease</a>, an incurable neurological condition, could continue. </p>
<p>The decision was based on the compassionate use of the therapy in patients with severe incurable diseases as a last resort. It also followed an official endorsement by the Italian Government in March 2013. The government decreed that the Stamina Foundation were allowed to continue stem cell therapy on 32 terminally ill patients. Their permission came without proof that the cell-based therapies were manufactured according to Italy’s legal safety standards. </p>
<p>After enormous criticism from Italian <a href="http://www.eurostemcell.org/files/Italian_researchers_letter_to_Ministry_of_Health_English.pdf">stem cell scientists</a>, the treatments were <a href="http://blogs.nature.com/news/2013/10/minister-halts-italian-stem-cell-therapy-trial.html">halted</a> by the Italian Ministry of Health in September 2014.</p>
<p>In <a href="http://dij.sagepub.com/content/48/6/681.full.pdf+html">Japan</a>, the revised Pharmaceutical, Medical Devices and other Therapeutic Products Act came into effect in November 2013. It contains a specific section for regenerative medical products and allows for conditional, time-limited marketing authorisation to be given. The authorisation will only happen if a product’s safety is ensured. </p>
<p>The available clinical trial results should also predict likely efficacy. But the authorised products will still be subject to further safety and efficacy tests between conditional and final approval.</p>
<h2>Covering South Africa’s landscape</h2>
<p>Stem cell therapy holds the possibility of curing a large number of diseases. However, almost all of these therapies are still in early development and clinical trial stages. </p>
<p>Access to these therapies prior to regulatory approval might not only relieve human suffering, but will also legally enable scientists to test and prove their efficacy via an alternative means (other than in the clinical trial setting). </p>
<p>Although the benefits from the developer’s perspective are apparent, the patient’s safety and best interest must remain top-of-mind. Most importantly, the expectations of potentially vulnerable patients should be managed sensitively and accordingly. </p>
<p>The universal nature of stem cells therapy begs for harmonised international regulation, and South Africa can learn and benefit from current global developments. </p>
<hr>
<p><em>This article based on a paper published in a special South African Medical Research Council flagship edition of the South African <a href="http://hmpg.co.za/index.php/sajbl/issue/view/497/showToc">Journal</a> of Bioethics and Law.</em></p><img src="https://counter.theconversation.com/content/45927/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Marco Alessandrini has received funding from National Research Foundation (NRF) of South Africa and the National Health Laboratory Services (NHLS) Trust.</span></em></p><p class="fine-print"><em><span>Marietjie Botes does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.</span></em></p>Many countries are introducing legislation and allowing practitioners to use medication still undergoing trials but that show preliminary signs of being safe, including some stem cell treatments.Marietjie Botes, PhD Student in Biotechnology Law, University of PretoriaMarco Alessandrini, Consultant, Cell Therapy and Genomics, University of PretoriaLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/454982015-08-20T04:31:53Z2015-08-20T04:31:53ZWhy South Africa needs better laws for stem cell research and therapy<figure><img src="https://images.theconversation.com/files/92439/original/image-20150819-10868-1jd2932.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Legislation in South Africa needs to be updated to accommodate the development in stem cell research and therapies.</span> <span class="attribution"><span class="source">Reuters</span></span></figcaption></figure><p><em>This article is part of a series The Conversation Africa is running on stem cell research and therapy. Read the rest of the series <a href="https://theconversation.com/au/topics/african-stem-cells">here</a>.</em></p>
<p>South Africa has a patchy array of laws dealing with stem cells. While it does have legislation in place, like many other countries its laws have failed to keep pace with developments in science.</p>
<p>Many countries have implemented strict legislation that will govern how stem cells can be used. If South Africa wants to be a competitive player in the global stem cell research and therapy sphere, it must amend its laws to provide updated, modern and unambiguous legislation. The legislation must have appropriate context, detail and clarity to stipulate what is allowed and what is not.</p>
<p>Stem cells are classified as biological medicines since they have been derived from living organisms or tissues. They have been used for therapeutic purposes for several decades in South Africa and abroad. Recently research in the stem cell field in South Africa has seen a marked increase which is likely to be sustained. </p>
<p>South Africa has a National Health Act which provides a legal framework for the use of <a href="http://www.chr.up.ac.za/undp/domestic/docs/legislation_55.pdf">human tissues</a>, including stem cells. It also has the Medicines Control Act, which defines the application of this framework in the clinical setting. </p>
<p>But the current legislation is not adequate. There are several important areas which remain unregulated. There is also a lack of harmonisation between the different pieces of legislation. And some of the definitions related to the technology are frankly incorrect. </p>
<h2>Adult stem cells</h2>
<p>Adult stem cells, which include those derived from the bone marrow, are regulated principally by Chapter Eight of the <a href="http://www.chr.up.ac.za/undp/domestic/docs/legislation_55.pdf">National Health Act</a>. These are regularly used in bone marrow transplants for diseases such as leukaemia and myeloma. </p>
<p>The existing legislation covers the harvesting, storage, import and export and processing of stem cells.</p>
<p>But more detail is needed in the form of specific guidelines on harvesting, storage and processing as well as the clinical application of these cells.</p>
<h2>Embryonic stem cells</h2>
<p>Embryonic stem cells, which are harvested from a five-day-old embryo in a laboratory and can develop into any one of the 200 cell types in the body, are governed principally by sections of the National Health Act dealing with the human embryo. According to the act, the derivation and use of these cells is permitted only with ministerial authorisation. </p>
<p>Because they can develop into all the body’s cell types, embryonic stem cells have the potential to be used for the treatment of many diseases. Currently they are used in research and for drug screening. There are, however, a few clinical trials underway aimed at assessing the usefulness of these cells for the <a href="https://clinicaltrials.gov/">treatment of blindness</a>.</p>
<p>Because human embryonic stem cells are obtained from a five-day-old embryo, and since this results in the destruction of the embryo, the harvesting of these cells has met with strong objections on religious, moral and ethical grounds.</p>
<p>Embryonic stem cells can be derived by several means – one of which requires fertilisation, also known as conception. The existing legislation covers this area. But another way to create these cells is by a process called somatic cell nuclear transfer, which does not require fertilisation. </p>
<p>Since the definition of an embryo in the National Health Act is “a human offspring in the first eight weeks from conception”, embryonic stem cells produced by a process which does not require conception are not regulated.</p>
<h2>Induced pluripotent stem cells</h2>
<p>Current legislation does not cover <a href="http://www.nature.com/scitable/topicpage/turning-somatic-cells-into-pluripotent-stem-cells-14431451">induced pluripotent</a> stem cells, which are derived using a groundbreaking technology in which adult stem cells are reprogrammed to an embryonic stem cell-like state. </p>
<p>To date, <a href="http://stemcellassays.com/2015/08/cells-weekly-august-2-2015/">induced pluripotent</a> stem cells have been created from a wide range of cell types and differentiated into an equally broad range of cell types. These stem cells are used to understand disease processes and for drug discovery. One clinical trial has been initiated in Japan for the treatment of blindness, but this has been temporarily suspended.</p>
<p>Among the cell types that can be derived from induced pluripotent stem cells are gametes (egg and sperm). How this will be regulated is not clear, although the legislation does cover some aspects of gametes per se. </p>
<h2>Other challenges</h2>
<p>In addition to the legislative challenges around stem cells, there is the phenomenon of gene editing. Gene editing, which began more than a decade ago, involves changing the DNA of a cell in a <a href="http://www.sciencemag.org/content/346/6213/1258096?intcmp=collection-crispr">laboratory</a>. The idea behind the modification is to be able to remove or repair a mutation in the cells of people with genetic and other diseases including HIV, cystic fibrosis and immunodeficiencies, among many. </p>
<p>One of the limitations in the process of gene editing is the unknown degree of “off-target” <a href="http://www.nature.com/nbt/journal/v33/n2/full/nbt.3127.html">effects</a>, where in addition to the modification of the gene interest, other unintended sites in the genome are also modified. This may have negative consequences. </p>
<p>Gene editing is the focus of great deal of <a href="http://www.nature.com/news/don-t-edit-the-human-germ-line-1.17111">debate</a> globally particularly in the context of <a href="http://www.ncbi.nlm.nih.gov/pubmed/25894090">germline engineering</a>. The debate has resulted in scientists across the globe proposing a moratorium on germline editing in humans until the implications of this type of <a href="http://www.sciencemag.org/content/348/6230/36">research</a> are better understood.</p>
<p>In the South African context, there are only a limited number of <a href="http://www.nature.com/mt/journal/v21/n10/full/mt2013170a.html">researchers</a> that are applying gene editing in the laboratory for [<a href="http://www.sciencedirect.com/science/article/pii/S0092867413012257">research purposes</a>. </p>
<p>From a global perspective, there is virtually no legislation governing the application of this new technology in the clinical setting. It would however be good to initiate a debate on this topic in the country.</p>
<h2>Improving stem cell regulation</h2>
<p>In South Africa, patients are protected from possible harm from medical treatments including stem cell therapy through the rights enshrined in the Constitution. But detailed legislation defining the scope, limits and requirements for the everyday practice in the field of stem cell research and therapy is limited. </p>
<p>In addition, South African law in general does not address the problem of stem cell tourism, where bogus treatments are applied to vulnerable patients. </p>
<p>There is an urgent need to establish a national oversight and regulatory body to provide guidance on the legal regulation of stem cell research and cell-based therapy. This body would provide timely and effective input on matters that often require a prompt legislative response.</p>
<p>And although many of the country’s health practitioners engage in self-regulation, formal endorsement from the National Department of Health of the guidelines that have been established is needed.</p>
<hr>
<p><em>This article based on a paper published in a special South African Medical Research Council Flagship edition of the South African <a href="http://hmpg.co.za/index.php/sajbl/issue/view/497/showToc">Journal</a> of Bioethics and Law.</em></p><img src="https://counter.theconversation.com/content/45498/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Michael Sean Pepper receives research funding from the South African Medical Research Council, The National Research Foundation of South Africa and the National Health Laboratory Services Research Trust.</span></em></p><p class="fine-print"><em><span>Janine Scholefield receives parliamentary grant funding and is supported by the Department of Science and Technology</span></em></p><p class="fine-print"><em><span>Melodie Slabbert works for the College of Law, University of South Africa.</span></em></p><p class="fine-print"><em><span>Robea Ballo receives funding from the South African Medical Research Council and the National Research Foundation of South Africa.</span></em></p>South Africa may have legislation broadly guiding stem cell research and treatment, but these laws must be updated and clarified for it to be effective.Michael Sean Pepper, Director of the Institute for Cellular and Molecular Medicine, University of PretoriaJanine Scholefield, Senior Scientist, Council for Scientific and Industrial ResearchMelodie Labuschaigne, Deputy Executive Dean and Professor of Law, College of Law, University of South AfricaRobea Ballo, Lecturer and Stem Cell Researcher, Division of Cell Biology, Department of Human Biology, University of Cape TownLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/443342015-08-19T04:43:34Z2015-08-19T04:43:34ZWhat lies behind the hype and the hope of stem cell research and therapy<figure><img src="https://images.theconversation.com/files/92289/original/image-20150818-12454-lg9bh7.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Embryonic stem cells. </span> <span class="attribution"><span class="source">shutterstock</span></span></figcaption></figure><p><em>This article is part of a series The Conversation Africa is running on stem cell research and therapy. Read the rest of the series <a href="https://theconversation.com/au/topics/african-stem-cells">here</a>.</em> </p>
<p>The words “stem cell research and therapy” evoke a number of responses. In emotionally vulnerable patients, a sense of hope. In scientists, a great deal of excitement about future prospects. In the case of legal experts and ethicists, a need to ensure that patient safety and a spirit of distributive justice are maintained. And in the minds of entrepreneurs, an opportunity to develop a profitable business.</p>
<p>Stem cells are the building blocks of our bodies. They are able to differentiate into the more that 200 cell types that make up our bodies. From a fertilised egg to a fully fledged human being which contains billions of cells, the purpose of stem cells during development in the womb is to ensure normal structure and function.</p>
<p>In postnatal life, stem cells replace those cells that have been damaged by wear and tear or by disease. </p>
<h2>Gaining momentum</h2>
<p>In research, stem cells are at the cutting edge of science, with regular breakthroughs being announced in the field. By 2012, it was estimated that there were close to <a href="http://www.eurostemcell.org/files/Stem-Cell-Report-Trends-and-Perspectives-on-the-Evolving-International-Landscape_Dec2013.pdf">100,000</a> active stem cell researchers across the globe. Massive <a href="http://stemcells.nih.gov/research/funding/pages/Funding.aspx">funding</a> is being directed globally into research which continues to provide hope to millions of patients.</p>
<p>Stem cell therapy translates the research findings into potential cures for many diseases. For instance, for more than 50 years, <a href="http://www.eurostemcell.org/files/Stem-Cell-Report-Trends-and-Perspectives-on-the-Evolving-International-Landscape_Dec2013.pdf">bone marrow transplants</a> – also known as hematopoietic stem cell transplants – have been used to treat patients with blood cancers such as <a href="http://www.medicinenet.com/leukemia/article.htm">leukemia</a> and blood disorders such as <a href="http://www.nhlbi.nih.gov/health/health-topics/topics/sca">sickle cell disease</a> and <a href="http://www.healthline.com/health/thalassemia">thalassemia</a>. </p>
<p>When a person with cancer undergoes conditioning chemotherapy to destroy the cancerous cells in the body, in the process this treatment also destroys the patient’s own stem cells. Bone marrow transplants are used to replace these stem cells. This form of treatment is universally employed, and accepted.</p>
<p>More recently, skin grown from stem cells has been used to treat extensive burns and stem cells from fat (adipose tissue) have been used as tissue fillers.</p>
<h2>The reality of stem cells versus future promise</h2>
<p>Stem cell treatment has saved many lives. But there are also elements of stem cells that have been mired in controversy.</p>
<p>As a result of stem cells becoming a buzzword, there has been a proliferation of websites offering <a href="http://www.eurostemcell.org/commentanalysis/stem-cell-tourism-selling-hope-through-unproven-stem-cell-treatments-lessons-x-cell-">dubious treatments</a>, luring people with incurable diseases who are emotionally vulnerable. There is rarely any form of control over what these clinics place on their websites, let alone the treatments they offer. </p>
<p>Aside from bone marrow transplants and stem cells used for burns, almost all other conditions for which stem cells are advertised to provide a cure are still in an experimental stage. Globally, there are hundreds of legitimate clinical <a href="https://clinicaltrials.gov/">trials underway</a> to assess the effect of stem cells in a variety of conditions including heart disease, spinal cord injury, blindness and Parkinson’s disease, to name a few. </p>
<p>But, in these cases, the road which finally joins the healing properties of stem cells to the approved use of these cells on a routine basis is long and arduous.</p>
<p>Clinical trials need to be undertaken before a treatment can become part of routine medical practice. They must be registered with the relevant national body in the country where they are taking place. Clinical trials also need to be peer reviewed via a registered ethics committee or an institutional review board. </p>
<p>And although rarely mentioned explicitly in legislation or guidelines, patients who receive experimental treatments should not have to pay for these treatments. </p>
<h2>Breaching the law on multiple fronts</h2>
<p>For most stem cell treatments which have not undergone clinical trials, patients are subjected to therapy which defies the basic ethical and legal principles of the medical profession. Some treatments are blatantly unsafe, such as the infusion of embryonic and animal-derived stem cells into <a href="http://www.eurostemcell.org/resource/tumour-formation-embryonic-stem-cells">humans</a>.</p>
<p>But practitioners who provide these unproven treatments argue that:</p>
<ul>
<li><p>patients are desperate and it is a last resort after trying everything else;</p></li>
<li><p>if one uses the patient’s own cells the rules do not apply; and </p></li>
<li><p>patients should have the right to decide how they wish to use their cells.</p></li>
</ul>
<p>Countries without adequate legislation cannot curb unethical practices and financial exploitation of patients using unproven stem cell treatments. In these countries, unscrupulous medical practitioners providing these therapies often identify the gaps in the law and then head straight for them, using legal tactics and devious interpretations to justify their activities. </p>
<h2>Regulating stem cell treatment</h2>
<p>To ensure the safety of stem cell treatments and to limit exploitation of vulnerable patients, several measures can be undertaken. These include establishing appropriate legislation, ensuring that this legislation is enforced, and educating the public.</p>
<p>Ethical advertising standards also need to be enforced to limit the dissemination of false information. And patients should feel they have the freedom to approach their medical practitioners for advice on how to proceed.</p>
<p>Without an adequate legislative environment or the enforcement of existing legislation, the medical industry is at risk of facing legal challenges from unsatisfied or damaged patients. This is likely to slow down advances in the field, although it will also provide much needed case law which, due to the relative youth of the field, is still lacking in many countries, including South Africa.</p>
<p>But the outcome could also include a knee-jerk reaction that results in excessively prescriptive legislation that limits research on valuable ethically and scientifically approved projects as well as the translation of research findings into useful products and services.</p>
<p><em>This article based on a paper published in a special South African Medical Research Council Flagship edition of the <a href="http://hmpg.co.za/index.php/sajbl/issue/view/497/showToc">South African Journal of Bioethics and Law</a>.</em></p><img src="https://counter.theconversation.com/content/44334/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Michael Sean Pepper receives funding from the South African Medical Research Council, the National Research Foundation of South Africa and the National Health Laboratory Services Research Trust.</span></em></p><p class="fine-print"><em><span>Nicolas Novitzky receives funding from the National Research Foundation and the Medical Research Council and he is a board member of the South African Bone Marrow Registry.</span></em></p>Stem cell research and therapy have become buzzwords across the globe. Although some of the treatments are controversial and unsafe, there is also a great deal of excitement and promise.Michael Sean Pepper, Director of the Institute for Cellular and Molecular Medicine, University of PretoriaNicolas Novitzky, Professor of Haematology, University of Cape TownLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/455022015-08-19T04:43:19Z2015-08-19T04:43:19ZA beginner’s guide to understanding stem cells<figure><img src="https://images.theconversation.com/files/92259/original/image-20150818-12428-1p8j0os.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Human bone marrow stem cells. </span> <span class="attribution"><span class="source">Shutterstock</span></span></figcaption></figure><p><em>This article is part of a series The Conversation Africa is running on stem cell research and therapy. Read the rest of the series <a href="https://theconversation.com/au/topics/african-stem-cells">here</a>.</em></p>
<p>Broadly speaking, <a href="http://stemcells.nih.gov/info/basics/pages/basics1.aspx">stem cells</a> are used to treat disease or repair damaged tissue, to understand disease processes and for drug discovery. They are able to be used for these purposes because they belong to a special group of cells that are capable of differentiation. This means that they can form any of the more than 200 different cell types found in our bodies.</p>
<h2>Different kinds of stem cells</h2>
<p>Although not a stem cell per se, the fertilised egg creates all the cells that make up the embryo and the placenta. There are two types of stem cells: <a href="http://www.medicinenet.com/script/main/art.asp?articlekey=14525">pluripotent</a> and <a href="http://stemcells.nih.gov/info/basics/pages/basics4.aspx">adult stem cells</a>. </p>
<p>Pluripotent stem cells are those that have the ability to form all the cells and tissues in the body (excluding the placenta). They are classified into either <a href="http://stemcells.nih.gov/info/basics/pages/basics3.aspx">embryonic stem cells (ESCs)</a> or <a href="http://stemcells.nih.gov/info/basics/pages/basics10.aspx">induced pluripotent stem cells (iPSCs)</a>. </p>
<p>While ESCs are derived from the early embryo, induced pluripotent stem cells are created when adult stem cells are reprogrammed to become like ESCs. By culturing adult cells in the laboratory in the presence of genes which are functional in the early embryo, the adult genes are switched off and the cells’ embryonic genes switched on.</p>
<p>As our bodies develop, cells become more restricted in their capacity to differentiate into other cells types, and are termed multipotent or unipotent. After we are born, adult stem cells replace cells lost through normal wear-and-tear or disease.</p>
<p>Adult stem cells are found throughout our bodies. There are several types. For example, hematopoietic (blood) stem cells are found in the bone marrow. They give rise to red blood cells, white blood cells and platelets. Another example are neural stem cells found in the nervous system. Mesenchymal stem cells are found in fat (adipose) tissue, bone marrow and the umbilical cord. </p>
<h2>What can stem cells be used for?</h2>
<p>Adult stem cells have been used for more than five decades to treat certain blood cancers and genetic or immunological disorders. Known as bone marrow or hematopoietic stem cell transplantation, this procedure replaces the normal stem cells in a patient’s body that have been destroyed by high dose chemotherapy.</p>
<p>Stem cells are also used to replace skin in major burn injuries and to heal chronic wounds. A wide-range of other potential uses are being tested but at this stage are considered to be experimental. These include treatment for heart disease, cerebral palsy, Alzheimer’s and Parkinson’s diseases, diabetes and spinal cord injury.</p>
<p>Adult stem cells are collected on a routine basis in many parts of the world. Current sources include the bone marrow, circulating blood, and cells harvested from umbilical cord blood. Adult stem cells, and in particular those derived from umbilical cord blood, can be stored for future use either in a public or a private cord blood bank.</p>
<p>Pluripotent stem cells are not yet used to treat patients routinely but are being tested in clinical trials for a number of diseases including blindness. At present they are mainly used to understand disease processes and for drug discovery.</p>
<h2>Why are stem cells controversial?</h2>
<p>More than 90% of the work involving stem cells is not controversial. This includes the therapies that are legitimately administered every day as well as most of the research work being done in many parts of the world. </p>
<p>Nonetheless, there are a few areas in the stem cell field that have become controversial. These include:</p>
<ul>
<li><p>embryonic stem cells, whose preparation is seen to result in the destruction of a potential life. This is because ESCs are derived from a small group of cells in a five day embryo (called the inner cell mass) that under normal circumstances would go on to form an entire living organism; </p></li>
<li><p><a href="http://www.womens-health.co.uk/stem_cell_ethics.html">reproductive cloning</a> in which a clone or copy of an organism is produced from a single cell removed from that organism; this is the process that produced Dolly the sheep. Reproductive cloning is universally banned in humans; and </p></li>
<li><p>stem cell tourism, where patients pay large amounts for unproven stem cells therapies, contravening ethical norms and safety standards.</p></li>
</ul>
<p><em>This article based on a paper published in a special South African Medical Research Council Flagship edition of the <a href="http://hmpg.co.za/index.php/sajbl/issue/view/497/showToc">South African Journal of Bioethics and Law</a>.</em></p><img src="https://counter.theconversation.com/content/45502/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Michael Sean Pepper receives research funding from the South African Medical Research Council, The National Research Foundation of South Africa and the National Health Laboratory Services Research Trust.</span></em></p>Stem cell research and therapy has taken the world by storm. Here’s what you need to know about it.Michael Sean Pepper, Director of the Institute for Cellular and Molecular Medicine, University of PretoriaLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/399432015-04-14T12:26:29Z2015-04-14T12:26:29ZCuring baldness may just be about having enough pluck<figure><img src="https://images.theconversation.com/files/77517/original/image-20150409-15216-1c8pjzr.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Ok, when will it come back?</span> <span class="attribution"><a class="source" href="http://i.huffpost.com/gen/1294515/images/o-BALD-facebook.jpg">HuffingtonPost.com </a>, <a class="license" href="http://creativecommons.org/licenses/by/4.0/">CC BY</a></span></figcaption></figure><p>Shaved heads have come in and out of fashion over the past few decades, but some people don’t have the option of allowing their locks to grow. Thankfully, for those who do suffer from hair loss, or alopecia, help may be at hand. Somewhat counter-intuitively an effective treatment for baldness may come from plucking a certain number of hairs – in a specific formation – from the scalp. </p>
<p><a href="http://www.nejm.org/doi/full/10.1056/NEJM199908123410706">Hair follicles</a> – the skin organ responsible for hair growth – contain stem cells that constantly divide, they are the driving force behind new hair growth. A healthy hair follicle produces about six inches of hair every year, but if the follicle stem cells malfunction and stop dividing, hair growth ceases and conditions such alopecia are observed. </p>
<p>Androgenic alopecia – or male pattern baldness – is the most common form of hair loss and will effect around two-thirds of men and one-third of women during their lifetime. </p>
<h2>Regeneration response</h2>
<p>Our <a href="http://www.cell.com/cell/abstract/S0092-8674%2815%2900182-8">recent study</a>, published in Cell, and completed on a mouse model, is unique because it not only studies the regeneration of a single hair follicle, but focuses on the regrowth of several follicles that had previously been effected by alopecia. </p>
<p>We demonstrated that plucking a few properly arranged hairs can trigger regeneration of hair follicles stem cells in up to five times more neighbouring, un-plucked surrounding hairs.</p>
<p>It is not surprising that follicle stem cell injury – caused by plucking – can cause a regeneration response. But, generally the stimulation of one stem cell through injury is only thought to cause regeneration in that stem cell alone. Triggering the regeneration of a whole head of hair in this way would be highly inefficient. But can the regeneration response of several stem cells be triggered by stimulating only a few key cells or signals? </p>
<h2>Decision making in stem cell populations</h2>
<p>Recently, we accidentally discovered that regeneration could occur through a collective decision-making process. By plucking the correct number of hairs with a proper arrangement, up to five times more neighbouring, unplucked resting hairs were activated to regrow. But if the number of plucked hairs was below a threshold, no hairs regenerated. </p>
<figure class="align-right zoomable">
<a href="https://images.theconversation.com/files/77519/original/image-20150409-15223-1g3lzxz.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/77519/original/image-20150409-15223-1g3lzxz.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/77519/original/image-20150409-15223-1g3lzxz.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=638&fit=crop&dpr=1 600w, https://images.theconversation.com/files/77519/original/image-20150409-15223-1g3lzxz.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=638&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/77519/original/image-20150409-15223-1g3lzxz.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=638&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/77519/original/image-20150409-15223-1g3lzxz.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=801&fit=crop&dpr=1 754w, https://images.theconversation.com/files/77519/original/image-20150409-15223-1g3lzxz.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=801&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/77519/original/image-20150409-15223-1g3lzxz.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=801&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
<figcaption>
<span class="caption">Collective-decision time.</span>
<span class="attribution"><a class="source" href="http://www.eurekalert.org/multimedia/emb/89363.php">Cheng-Ming Chuong</a>, <a class="license" href="http://creativecommons.org/licenses/by/4.0/">CC BY</a></span>
</figcaption>
</figure>
<p>This type of regeneration is an all-or-nothing process which is dependent on the signals produced by a fraction of hairs being plucked, and is an example of the process known as <a href="https://www.nottingham.ac.uk/quorum/what.htm">“quorum sensing”</a>. </p>
<p>Quorum sensing can be thought of as a decision-making process which is dependant on certain criteria being met within a population. Signalling molecules are released by each stimulated component of the population, the more components that are stimulated the more signal molecules are released. As the elements in the system are able sense the number of signal molecules released by the population as a whole, they can also sense the degree of stimulation. When a certain threshold of stimulation is reached, a collective response from the components in the system will follow. </p>
<p>The process of quorum sensing has been used to describe bacteria cell-to-cell communication, where the expression of certain genes is coordinated between many bacteria in response to environmental factors such as an increase in the presence of bacterial toxins. Quorum sensing has also been used successfully to explain the <a href="http://jackknife.med.yale.edu/nsci590-2009/pdfs/pratt2002.pdf">behaviour of social insects</a> such as ants and honey bees for their collective decision-making.</p>
<h2>Cast and count</h2>
<p>But in reality, how does the population of hair follicles “cast and count its vote” in quorum sensing?. First, there is a stimulus – such as hair plucking, which stimulates follicle stem cells – to some, but not all, hair follicles. Second, the plucked hair sends out a signal to surrounding cells. Third, the group of cells gauges the intensity of signal from its surroundings. Finally, a local decision is made within the population in an all-or-nothing fashion: if enough hairs have been plucked, mass hair regrowth will occur, but if not, there will be no response at all. </p>
<p>In the most simple cases of quorum sensing, the signal molecule spreads by diffusion from the secreting cell. But it was found that the signals being released by plucked hair follicles were travelling further than could be achieved by simple diffusion, suggesting that a something more complicated was involved. </p>
<p>Molecular and genetic analysis revealed that the signals were transmitted through a two-step immune response, triggered by the plucking of the hair follicle. First injured hair follicle stem cells will release a small signal molecule, this recruits a specific cell type involved in the immune response called a <a href="http://www.nature.com/nri/focus/macrophages/index.html">macrophage</a>. This then secretes a signal molecule involved in the immune response called a <a href="http://www.news-medical.net/health/What-are-Cytokines.aspx">cytokine</a>, which acts directly on surrounding hair follicle cells by stimulating various cellular regeneration signal pathways. </p>
<h2>Repair and regeneration</h2>
<p>This work shows that a quorum-sensing system can sense cell injury and use immune response to quantify how much damage has occurred. The stem cell population then disregards the stimulus if the minimum number of hairs has not been plucked, or responds to it with a full-scale regenerative response in many hair follicles when a threshold is reached. </p>
<p>This finding also is important in the field of regeneration medicine as a whole. We believe that the quorum-sensing behaviour principle is likely to be present in the regeneration of tissue and organs beyond the skin. Using such efficient regenerative strategies opens a new window in treating hair loss as well as many other degenerative disorders.</p>
<p><strong><em>This article was co-written with Chih-Chiang Chen from the Taipei Veterans General Hospital</em></strong></p><img src="https://counter.theconversation.com/content/39943/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Cheng-Ming Chuong has receive funding from National Institutes of Health (NIH)</span></em></p>Plucking hairs from the scalp causes surrounding hairs to regenerate, and could be a novel treatment for alopecia sufferers.Cheng-Ming Chuong, Professor of Tissue Development and Regeneration, University of Southern CaliforniaLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/277312014-06-23T05:05:57Z2014-06-23T05:05:57Z‘One of Us’ petition marks a sinister mobilisation of the pro-life movement in Europe<figure><img src="https://images.theconversation.com/files/51657/original/6hxcv42v-1403175551.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">One of us: an exclusive club.</span> <span class="attribution"><a class="source" href="https://www.flickr.com/photos/lilongd/5627197631/sizes/l/in/photolist-9zfRNP-g5QkGF-8faC9b-9kCVBV-8E7ChA-bheo7a-9ePNaj-88WZV9-7X1XUC-dbqu4V-cskfgQ-dPhRVp-wmmfW-9Fhzcf-5yY9Sr-ejGr7Q-6tzuft-4ym6Gt-9N7nr8-aF7qr6-6ubx2s-5HVNqb-wBC9c-4Mt8dp-8frxSf-aK8Qgt-5AXtkS-fe3WvH-jZmwrE-8mbr5L-aApyDi-fzKLAt-iiyfT-AeEcr-aurEYh-bRNLtx-3jVPDK-fr5Z2g-iZJ9e-8zcdNj-8XTmfL-6L7pbV-2SmfSo-hTZ2xz-6o8UHa-nmWQg5-k4oqaT-9XiLqb-hBu5d-bZqqQy-8YafFR/">Lilongd</a>, <a class="license" href="http://creativecommons.org/licenses/by-sa/4.0/">CC BY-SA</a></span></figcaption></figure><p>In the European Union, the European Citizen’s Initiative was set up with the aim of bringing the activities of the European Parliament closer to the citizens of Europe. If a petition can achieve 1m signatures from across at least seven member states, the European Commission, which devises common policies, is obliged to consider the proposal and a legal framework to allow it to happen.</p>
<p>One of the most successful of these ECIs achieved the requisite 1m signatures late last year under the tagline of “a beautifully simple step but could help save millions of lives.” But this ECI was anything but beautiful and <a href="http://www.euractiv.com/sections/development-policy/uk-warns-meps-against-evangelical-attack-eu-development-aid-301614">threatened to endanger the lives</a> of thousands of women worldwide. </p>
<p>As we wrote, when it first <a href="http://www.birmingham.ac.uk/news/thebirminghambrief/items/2012/10/Eroding-womens-basic-freedoms-Current-trends-to-reduce-access-to-abortion.aspx">appeared in 2012</a> the ECI said: </p>
<blockquote>
<p>The human embryo deserves respect to its dignity and integrity. This is enounced by the European Court of Justice in the Brüstle case, which defines the human embryo as the beginning of the development of the human being. To ensure consistency in areas of its competence where the life of the human embryo is at stake, the EU should establish a ban and end the financing of activities which presuppose the destruction of human embryos, in particular in the areas of research, development aid and public health.</p>
</blockquote>
<p>If successful the ECI had the potential to severely restrict human embryonic stem cell research within the EU. But more worrying is the impact that it could have had on the lives of women in countries in receipt of EU development aid; the countries where <a href="http://gamapserver.who.int/maplibrary/files/maps/global_mmr_2013.png">maternal mortality and morbidity are higher</a> than those we consider acceptable within the EU, and where access to safe and legal abortion <a href="http://unfpa.org/public/home/sitemap/icpd/international-conference-on-population-and-development/icpd-summary">is a basic health need</a>. </p>
<p>If successful it would have directly challenged fundamental rights of women and been in direct conflict with the aims of UN Millennium Development Goal five: <a href="http://www.un.org/millenniumgoals/maternal.shtml">to improve maternal health</a>.</p>
<p>“One of Us”, the name of the campaign behind the proposal, was able to harness the infrastructure of the Roman Catholic Church, complete <a href="http://www.oneofus.eu/pope-benedict-xvi-supports-the-one-of-us-campaign-in-favour-of-human-life/">with an endorsement</a> by Pope Benedict XVI, to gather signatures from across ten member states. Other Christian groups also back the campaign.</p>
<h2>Weak legal basis</h2>
<p>In early April this year the co-ordinators of this ECI had the opportunity to present their arguments to Máire Geoghegan-Quinn and Andris Piebalgs, the EU commissioners for research and development aid repectively. A month later the commission officially responded to the ECI and – thankfully for women’s and human rights – rejected the premises upon which it was based and declined to consider a legislative proposal that might give effect to it.</p>
<p>As noted in the response, the legal basis for this ECI was weak from the start. It purported to draw from the decision in <a href="http://kslr.org.uk/blogs/europeanlaw/2012/07/13/oliver-brustle-vs-greenpeace-how-to-read-the-moral-compass/">the case of Oliver Brüstle v Greenpeace</a> in defining the human embryo as existing from the moment of conception for the purposes of legal protection. This case required the court to consider how embryos are defined in a commercial setting for the purposes of patenting and intellectual property.</p>
<p>But this claim deliberately overlooked the fact that the decision in the Brüstle case was concerned solely with the issue of patentability. As such it specifically did not, and was not an attempt to, provide a precedent for a more wide-ranging definition of legal personhood or serve as a comment on whether human embryonic stem cell research is permissible or something that should be funded.</p>
<h2>Europe’s very own ‘gag rule’</h2>
<p>“One of Us” is modelled on a restriction, introduced by the Ronald Reagan in 1984, often called “<a href="http://populationaction.org/topics/global-gag-rule/">the global gag</a>.” This is a prohibition on organisations that receive US government funding from facilitating access to abortion services or any advocates for the liberalisation of domestic abortion policy. This restriction applies even if the organisation provides a broad range of sexual and reproductive health services and obtains its funding for abortion services from another source. </p>
<p>The “global gag” has been endorsed by every Republican president since Reagan and rescinded by every Democratic president. The gag applies in countries where abortion is legal (neither US nor EU development aid is used to fund access to abortion where the procedure is illegal). The impact of the global gag <a href="https://www.guttmacher.org/pubs/tgr/04/3/gr040301.html">has been measured</a> by several organisations including the World Health Organisation, the Guttmacher Institute, and Population Action International and, unsurprisingly, they’ve found that it has increased the number of unplanned pregnancies and abortions because of the impact it has had on family planning and contraceptive care more generally. </p>
<p>It has also had significant and negative impact on the lives of real women in countries where access to safe abortion is a legal <a href="https://www.guttmacher.org/pubs/gpr/16/3/gpr160309.html">and necessary part</a> of abortion care.</p>
<p>Campaigns like One of Us are not a “beautifully simple step” to saving millions of lives. They are moves intended to disadvantage and marginalise women; sadly often those most in need of assistance – women in developing countries are “one of us” and should be treated as such.</p>
<p>It also highlights the potential negative impacts for which the seemingly benign citizen’s initiatives, like popular petitions, can be co-opted by other interested groups. Importantly it is a mechanism that some believe <a href="http://www.euractiv.com/sections/development-policy/uk-warns-meps-against-evangelical-attack-eu-development-aid-301614">will be used again</a> in the furtherance of conservative aims.</p>
<p>It was always likely that this initiative would fail from a legal perspective, however, a primary objective was not success <a href="http://www.oneofus.eu/faq-2/">but the creation</a> of “a new Europe-wide mobilisation of the pro-life movement.” It is for this reason that it’s important to pay attention to the success of the initiative – in its own terms even if it did not formally succeed. </p><img src="https://counter.theconversation.com/content/27731/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>The authors do not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and have disclosed no relevant affiliations beyond their academic appointment.</span></em></p>In the European Union, the European Citizen’s Initiative was set up with the aim of bringing the activities of the European Parliament closer to the citizens of Europe. If a petition can achieve 1m signatures…Sheelagh McGuinness, Birmingham Law Fellow, University of BirminghamHeather Widdows, John Ferguson Professor of Global Ethics, University of BirminghamLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/236872014-02-28T06:04:19Z2014-02-28T06:04:19ZBAMI trial might provide bone marrow answers, but it won’t teach us much about stem cells<figure><img src="https://images.theconversation.com/files/42686/original/ds5gy4ng-1393519895.jpg?ixlib=rb-1.1.0&rect=0%2C2%2C1000%2C616&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">More than just getting from A to B.</span> <span class="attribution"><span class="source">Shutterstock</span></span></figcaption></figure><p>One of the world’s largest clinical cell therapy trials has <a href="http://www.bartshealth.nhs.uk/media/press-releases/2014/2014/february/barts-health-leads-largest-ever-adult-stem-cell-heart-attack-trial/">begun to enroll</a> 3,000 heart attack patients, some of whom will have bone marrow cells extracted with a needle from their hip and fed into their heart using a catheter in their coronary arteries. </p>
<p>The <a href="http://clinicaltrials.gov/ct2/show/NCT01569178">BAMI trial</a> has €5.9m in funding from the European Commission and will be conducted in ten European countries. Enlisted patients will be randomly assigned into two groups: one group will receive the standard care given to heart attack patients while the other will get an added infusion of bone marrow cells.</p>
<p>A number of studies, <a href="http://www.nejm.org/doi/full/10.1056/NEJMoa060186">including one</a> in the New England Journal of Medicine <a href="http://eurheartj.oxfordjournals.org/content/28/24/2998.abstract">and another</a> in the European Heart Journal, have suggested that bone marrow cells could be beneficial to patients with heart disease. However, because these studies were too small to work out whether cell infusions affected patients’ survival, they instead focused on the extent of scar formation after a heart attack or the ability of the heart muscle to contract after cell infusion. </p>
<p>One commonly used surrogate measure is the cardiac ejection fraction, which measures the fraction of blood squeezed out by the heart during a contraction. A healthy rate ranges from 55% to 65%. Bone marrow cell infusion has been associated with a modest but statistically significant improvement in heart function. In 2012, a <a href="http://circ.ahajournals.org/content/126/5/551.long">comprehensive analysis</a> of 50 major studies with a combined total of 2,625 heart disease patients showed that cardiac ejection fraction in patients receiving these infusions was 4% higher than in control patients. </p>
<p>While the results were encouraging, the study was a retrospective analysis with patients who had varying treatments and endpoints. There also remain questions over 400 patients included in the analysis from trials showing benefits of bone marrow cell infusions that were conducted by controversial German cardiologist Bodo Strauer, who <a href="http://blogs.nature.com/news/2013/07/german-cardiologists-stem-cell-papers-attacked.html">some scientists have accused</a> of errors in research. </p>
<p>The new large-scale BAMI trial will be able to provide a more definitive answer to the efficacy of bone marrow cell infusions and address the even more important question: does this experimental treatment prolong the lives of heart attack patients?</p>
<h2>A hard cell</h2>
<p>Despite the impressive target of enrolling 3,000 patients, there is a problem with how the trial is being framed. The underlying premise of why bone marrow cells are thought to improve heart function is that the bone marrow contains stem cells which could potentially regenerate the heart. In media reports, the BAMI trial is portrayed as a study which will test whether <a href="http://www.bbc.co.uk/news/health-26273707">stem cells can heal broken hearts</a>, and a press release by Barts Health NHS Trust, which is leading on the trial, described the study as “the <a href="http://www.bartshealth.nhs.uk/media/press-releases/2014/2014/february/barts-health-leads-largest-ever-adult-stem-cell-heart-attack-trial/">largest ever adult stem cell</a> heart attack trial”. But the scientific value of the BAMI trial for stem cell research is questionable. </p>
<p>In 2013, a <a href="http://circ.ahajournals.org/content/127/19/1968">Swiss study reported the results</a> of treating heart attack patients with bone marrow cells. Not only did the study find no significant improvement of heart function with cell therapy, the researchers also reported that only 1% of the infused cells had clearly defined stem cell characteristics. The <a href="http://circ.ahajournals.org/content/127/19/1935.long">vast majority</a> of the infused bone marrow cells were a broad mixture of various cell types, including immune cells such as lymphocytes and monocytes. </p>
<p>Scientific studies have even cast doubts about whether any of the scarce stem cells in bone marrow can convert into beating heart muscle cells. A study published in 2001 suggested bone marrow cells injected into mouse hearts could differentiate into heart muscle cells, but the finding <a href="http://circ.ahajournals.org/content/127/19/1935.long">could not be replicated</a> in a subsequent study published in 2004. </p>
<p>If there are so few stem cells in the bone marrow and if the stem cells do not become cardiac cells, then how does one explain the improvements observed in the smaller studies? Researchers have proposed a variety of potential explanations, including the release of growth factors or proteins by bone marrow cells that are independent of their stem cell activity.</p>
<h2>The disease machine</h2>
<p>The success of modern medicine lies in its ability to isolate causal mechanisms of disease and design therapies which specifically target these mechanisms using rigorous scientific methods. Instead of using nebulous “fever tinctures” or willow bark, physicians now prescribe therapies with well-defined active ingredients such as paracetamol (acetaminophen) or aspirin. </p>
<p>Infusing heterogeneous bone marrow cell mixtures into the hearts of patients seems like a throwback to the era of mysterious herbal extracts containing a variety of active and inactive ingredients. </p>
<p>Even if the BAMI trial succeeds in demonstrating that infusion of bone marrow cell mixtures can prolong lives, then the scientific value of the results will still remain doubtful. We will not know whether the tiny fraction of stem cells contained in the bone marrow was responsible for the improvement or whether this effect was due to one of the many other cell types contained in the cell mixtures. </p>
<p>One could argue that it is irrelevant to know the mechanism of action as long as the infusions can prolong patient survival. But for any evidence-based therapy to succeed, it is essential for physicians to know how to dose or modify the therapy according to the needs of an individual patient. This won’t be possible if we don’t even understand how the treatment works. </p>
<p>We should also consider the impact of a negative result. If the BAMI trial fails to show improved survival, will the lack of efficacy be interpreted as a failure of stem cell therapy for heart disease? An alternate explanation would be that a negative result was due to infusing numerous cell types, most of which were not stem cells.</p>
<p>The ultimate test of a treatment’s efficacy is how it fares in controlled, large-scale trials. And these trials need to be grounded in solid scientific data and provide answers that can be interpreted in the context of scientifically sound mechanisms. The BAMI trial might provide an answer to the question of whether or not bone marrow cell infusions are efficacious in heart disease, but it will not teach us much about stem cells.</p><img src="https://counter.theconversation.com/content/23687/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Jalees Rehman has received research funding from the National Institutes of Health (NIH).</span></em></p>One of the world’s largest clinical cell therapy trials has begun to enroll 3,000 heart attack patients, some of whom will have bone marrow cells extracted with a needle from their hip and fed into their…Jalees Rehman, Associate Professor of Medicine and Pharmacology, University of Illinois ChicagoLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/232142014-02-19T05:48:25Z2014-02-19T05:48:25ZOpinions about scientific advances blur party-political lines<figure><img src="https://images.theconversation.com/files/41480/original/bnf79f3m-1392310283.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Science has come a long way, it's time for communication to catch up.</span> <span class="attribution"><span class="source">Pieter Kuiper</span></span></figcaption></figure><p>Reading about the rapid pace of advances in biomedicine, you may have wondered why more politically liberal countries like Germany and Canada have stronger restrictions on embryonic stem cell research than the politically conservative US.</p>
<p>History and happenstance play a role, but these differences also reflect public concerns that do not conform to traditional left or right political ideologies. </p>
<p>As debates over stem cell research continue and as conflicts over other biomedical advances emerge, a recurring set of questions is likely to be seen. Do scientific breakthroughs promote or undermine social progress? Is research being pursued too cautiously or too quickly? Do scientists respect or cross moral boundaries? </p>
<p>In a study, just published in <a href="http://dx.plos.org/10.1371/journal.pone.0088473">PLOS ONE</a>, we analysed a series of surveys collected between 2002 and 2010 to better understand what the US public thinks about stem cell research and how they formed these opinions. We were able to distinguish between the different factors influencing their beliefs. At play were factors such as traditional loyalties to political parties and more fundamental beliefs about science and society.</p>
<p>Our results indicate that, more than political party identification, ideology or religious beliefs, an individual’s beliefs about science and society had the strongest influence on their support for stem cell research. It was also possible to identify distinct segments who differ substantially in what they thought about science’s social implications. Traditional political labels do not easily define these groups.</p>
<p>Based on our data we classify the US public in four categories:</p>
<ol>
<li><p><strong>Scientific optimists</strong>: These comprise about a third of the public. They believe strongly in the link between science and social progress. They are likely to support most scientific advances and three quarters of this group are in favour of embryonic stem cell research. Optimists are on average highly educated, financially well off and disproportionately white. They are split almost evenly along political lines, with slightly more Democrats among them. In terms of political ideology, they are the most moderate in their outlook.</p></li>
<li><p><strong>Scientific pessimists</strong>:This group comprises just under a quarter of the public. They have strong reservations about the moral boundaries that might be crossed by scientists and believe science may lead to new problems. They are the most likely to oppose advances in biomedical research, with only 40% in favour of stem cell research. Compared to optimists, this group scores much lower on average in terms of educational attainment and income. More tend to be female and from a minority background. Pessimists split evenly along party lines, but tend to be disproportionately either moderate or conservative in their ideological outlook.</p></li>
<li><p><strong>The conflicted</strong>: This group represents another quarter of the public. They view science in both optimistic and pessimistic terms. Though they are socially similar to the Pessimists in their background, they tend to be older on average than members of other segments. They appear open to accepting the arguments of scientists and advocates who emphasise the benefits of research. By 2010, more than 60% of this segment had come to favour embryonic stem cell research.</p></li>
<li><p><strong>The disengaged</strong>: About 15% of the public lacks strong beliefs about how science might impact society. As a result, they are likely to be the most susceptible to shifts in opinion driven by political messages. For example, between 2008 and 2010, support for embryonic stem cell research among this group increased by 20 percentage points.</p></li>
</ol>
<h2>A better informed public</h2>
<p>As advances in stem cell research, synthetic biology, personalised genomics and other scientific fields move forward, our current media is unlikely to adequately address the deeper set of public concerns reflected in our study.</p>
<p>Cable TV, social media and the tabloid press tend to favour sensationalism over context. These complex debates are all too likely to be distorted in terms of simplistic left-right distinctions or exaggerated to be miracle breakthroughs or morally repugnant. At more prestigious news outlets, budget cuts and layoffs will limit the opportunity for in-depth coverage and analysis.</p>
<p>Given these challenges, academics need to invest in encouraging respectful debate about the future of science and what it means for society. The place to start may be in the cities and regions where research is taking place. Here we need to better understand the different questions being asked about scientific advances and invest in local media and public forums that encourage constructive discussion and debate.</p>
<p>But, given the international nature of science, we also need to think more broadly and ensure conversations transcend national boundaries. We need to take advantage of online media platforms to build what journalists Andrew Revkin and Krista Tippet have called the “<a href="http://dotearth.blogs.nytimes.com/2012/12/26/exploring-the-roots-of-an-emerging-planet-spanning-mind/">Knowosphere</a>,” a global classroom where interested people can learn about and discuss the social and ethical implications of science.</p>
<p>Revkin’s blog <a href="http://dotearth.blogs.nytimes.com/">Dot Earth</a> and Tippett’s multimedia series <a href="http://www.onbeing.org/">On Being</a> are prototypes for designing these new types of forums. So too is The Conversation. Yet more support for these forums and similar ventures are needed and it will ultimately be up to scientific institutions to lead the way.</p><img src="https://counter.theconversation.com/content/23214/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Matthew Nisbet has consulted on communication and public outreach activities with a range of life sciences organizations and has received speaking fees to address their scientists and staff. He currently serves as a member of the US National Academies Roundtable Committee on Public Interfaces in the Life Sciences. Funding for the study discussed in this article was provided exclusively by American University.</span></em></p><p class="fine-print"><em><span>Ezra Markowitz does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.</span></em></p>Reading about the rapid pace of advances in biomedicine, you may have wondered why more politically liberal countries like Germany and Canada have stronger restrictions on embryonic stem cell research…Matthew C. Nisbet, Associate Professor of Communication, American UniversityEzra Markowitz, Earth Institute Fellow, Columbia UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/186712013-10-03T13:45:40Z2013-10-03T13:45:40ZStem cells offer a more natural approach to plastic surgery<figure><img src="https://images.theconversation.com/files/32419/original/t8rtc9mg-1380801321.jpg?ixlib=rb-1.1.0&rect=0%2C0%2C1024%2C768&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Man in the mirror.</span> <span class="attribution"><span class="source">Shareski</span></span></figcaption></figure><p>The potential of stem cells is everywhere in medicine - from growing new tissue that could go on to provide replacement organs, repairing damage from disease or injury and in reconstructive surgery.</p>
<p>And a team of Danish researchers recently <a href="http://bit.ly/1fKCUW2">suggested in The Lancet</a> that stem-cell enriched fat grafts had the power to transform reconstructive surgery, including procedures such as breast surgery. Not only can they improve the survival of grafts, but also success of a more natural approach to reconstruction. </p>
<p>Stem cells are a class of undifferentiated cells that are able to become (differentiate into) specialised cell types, and they repair and replace cells in tissues. They have three vital characteristics: they are long-lived, they are self-renewing (we can make more of them) and can differentiate into more mature, specialised cells. Adult stem cells, also known as somatic stem cells, are found throughout the body and can multiply to regenerate damaged tissue or replace dying cells. They can be derived from many sources, including from fatty (adipose) tissue.</p>
<p>Stem cells have the potential to revolutionise plastic and reconstructive surgery, a speciality which focuses on restoring form and function. And introducing them to techniques in this field, such as fat grafting (otherwise known as lipofilling, where a patient’s own fat is harvested to increase its volume elsewhere in the body) could improve the outcome of the procedure and its longevity.</p>
<figure class="align-left ">
<img alt="" src="https://images.theconversation.com/files/32413/original/zp8bygkj-1380797349.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/32413/original/zp8bygkj-1380797349.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=600&fit=crop&dpr=1 600w, https://images.theconversation.com/files/32413/original/zp8bygkj-1380797349.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=600&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/32413/original/zp8bygkj-1380797349.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=600&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/32413/original/zp8bygkj-1380797349.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=754&fit=crop&dpr=1 754w, https://images.theconversation.com/files/32413/original/zp8bygkj-1380797349.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=754&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/32413/original/zp8bygkj-1380797349.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=754&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">A breast procedure without implants.</span>
<span class="attribution"><span class="source">Tips Times</span></span>
</figcaption>
</figure>
<p>Lipofilling is commonly performed in plastic surgery to replace lost volume. The procedure is used to improve breast symmetry following breast reconstruction surgery, augmentation and for congenital breast deformities without using breast implants. </p>
<p>But one of its drawbacks is the variation in how quickly the fat graft is reabsorbed by the body. Some studies have suggested <a href="http://cdn.intechopen.com/pdfs/33483/InTech-Autologous_fat_grafting_factors_of_influence_on_the_therapeutic_results.pdf">a gap as wide</a> as 20-80%. The Danish researchers in the Lancet suggest that by enriching these fat grafts with fat-derived stem cells, the amount of graft that survives absorption by the body is substantially improved.</p>
<p>While this has been shown in animal studies, the result in humans is a significant step forward for reconstructive medicine. It adds greatly to the prospect of stem cell use in clinical practice especially in breast reconstruction where large volumes of lipofilling are required. </p>
<p>It has the potential to significantly reduce the number of operative procedures a patient may require. It is also a step forward in other procedures where lipofilling is used such as filling in scars and traumatic defects.</p>
<h2>Nerve and burn injuries</h2>
<p>Stem cells are also being used tissue engineering and other areas of reconstructive medicine. For example, in patients with traumatic nerve injuries, artificial conduits are being used to guide new regrowth and have emerged as an alternative to nerve grafts taken from elsewhere in the body. Fat-derived stem cells have also been incorporated into these nerve conduits in recent experimental studies so that they differentiate into Schwann cells, which play and important role in conducting nerve impulses, to promote nerve regeneration.</p>
<p>There is a potential for the use of epidermal stem cells in burns. The stem cells can be sprayed on directly or transferred onto a scaffold of skin substitute or graft. This has been shown to <a href="http://www.ncbi.nlm.nih.gov/pubmed/21200267">improve wound healing</a>. In the same way, these stem cells have also been used to heal chronic wounds such as ulcers either as epidermal stem cells sprayed directly on to the wound or through fat-derived stem cells injected into the wound.</p>
<h2>Cosmetic procedures</h2>
<p>Cosmetic procedures and in particular facial rejuvenation has become an integral part of aesthetic plastic surgery. Many people are now opting to counter volume loss and wrinkles as they age. Synthetic fillers are used for this but they <a href="http://www.independent.co.uk/life-style/health-and-families/health-news/filler-injections-can-cause-permanent-damage-say-doctors-398943.html">have significant limitations</a>. One of the fundamental principles of plastic surgery is replacing like with like and using fat grafts from a patient’s own body is preferable. But the unpredictability of retention means that many still opt for a synthetic substance.</p>
<p>One must be aware, however, that studies show that stem cells do retain memory of their donor site. This has implications for cosmetically sensitive areas such as the area surrounding the eye should the patient gain weight. So if fat-derived stem cells were taken from the hip, for example, if the patient put on weight they might put on more where the graft is.</p>
<p>Claims are also being made that these cell therapies <a href="http://fabfitfun.com/stem-cells-in-skin-care">improve skin quality</a> and tightening. While this would be a boon for the beauty and cosmetics industry, there’s no concrete evidence for this.</p>
<p>There is no doubt that this is an important and exciting field. Stem cells look to become the corner stone of regenerative medicine. And in reconstructive surgery there is yet more to come.</p><img src="https://counter.theconversation.com/content/18671/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Ash Mosahebi works as a Consultant Surgeon for a number of NHS organisations as well as in private practice.</span></em></p>The potential of stem cells is everywhere in medicine - from growing new tissue that could go on to provide replacement organs, repairing damage from disease or injury and in reconstructive surgery. And…Ash Mosahebi, Honorary Senior Lecturer, UCLLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/151772013-06-21T04:30:51Z2013-06-21T04:30:51ZDream of regenerating human body parts gets a little closer<figure><img src="https://images.theconversation.com/files/25822/original/fmzkfcgw-1371619889.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">A new animal study shows we're making small progress in working out how to grow limbs.</span> <span class="attribution"><span class="source">Image from shutterstock.com</span></span></figcaption></figure><p>Damage to vital organs, the spinal cord, or limbs can have an enormous impact on our ability to move, function – and even live. But imagine if you could restore these tissues back to their original condition and go on with life as normal.</p>
<p>Well, this is the dream for regenerative medicine. And while humans missed out on these abilities in the evolutionary lottery, a <a href="http://www.nature.com/nature/journal/vaop/ncurrent/full/nature12214.html">recent study in mice</a> shows we’re making small progress to achieving this dream.</p>
<h2>Learning from animals</h2>
<p>Nature has provided the animal kingdom with many different ways to achieve perfect regeneration. Some amphibians – such as salamanders – are famous for their superhero-like ability to regenerate heart, brain, spinal cord, tail and can even whole limb tissue throughout their life. </p>
<p>Although organ and spinal cord regeneration are clinically important and worthy of intense research investment, regrowing whole limbs provides a flagship example of perfect regeneration in the salamander. </p>
<figure class="align-left ">
<img alt="" src="https://images.theconversation.com/files/25814/original/djmzf94p-1371617264.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/25814/original/djmzf94p-1371617264.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=400&fit=crop&dpr=1 600w, https://images.theconversation.com/files/25814/original/djmzf94p-1371617264.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=400&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/25814/original/djmzf94p-1371617264.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=400&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/25814/original/djmzf94p-1371617264.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=503&fit=crop&dpr=1 754w, https://images.theconversation.com/files/25814/original/djmzf94p-1371617264.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=503&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/25814/original/djmzf94p-1371617264.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=503&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">There’s no limit to the number of times lizard-like salamanders can regenerate their limbs.</span>
<span class="attribution"><span class="source">Image from shutterstock.com</span></span>
</figcaption>
</figure>
<p>It has been known for more than a hundred years that if a salamander loses a limb, it grows right back. This process is extremely precise and removal of the limb at the shoulder regrows a full limb, but removal at the wrist only regrows the missing hand portion. </p>
<p>Interestingly, there does not seem to be a limit on how many times they can perform this clever trick and each time the limb comes back perfect.</p>
<p>But mammals (including humans and mice) seem to have missed out on this important skill. The question of how to enhance the regenerative capabilities in humans, either by adding the missing ingredients, or activating these latent abilities currently lies wide open.</p>
<h2>Extending regeneration to mammals</h2>
<p>Mammals currently only have the capacity to regenerate the very tip of their finger. But the result is far from perfect. A range of studies in mice have shown the digit-tip regrowth is severely restricted. Removal of the very tip of the mouse digit will be replaced, but removal of the tissue a small distance further up the digit and closer to nail bed (the equivalent to a human cuticle), will fail to regrow. </p>
<p>Last week, a group of researchers from the United States and Japan <a href="http://www.nature.com/nature/journal/vaop/ncurrent/full/nature12214.html">published work</a> extending our understanding of the mechanism by which a resident stem cell population within the mouse digit tip nail bed can be activated to induce digit tip regeneration. In other words, we can now grow more of the digit back in mice and possibly more of the human finger. </p>
<figure class="align-right ">
<img alt="" src="https://images.theconversation.com/files/25821/original/t7kbpds8-1371619768.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/25821/original/t7kbpds8-1371619768.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=600&fit=crop&dpr=1 600w, https://images.theconversation.com/files/25821/original/t7kbpds8-1371619768.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=600&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/25821/original/t7kbpds8-1371619768.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=600&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/25821/original/t7kbpds8-1371619768.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=754&fit=crop&dpr=1 754w, https://images.theconversation.com/files/25821/original/t7kbpds8-1371619768.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=754&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/25821/original/t7kbpds8-1371619768.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=754&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">Like humans, mice can usually only regenerate the tip of their finger.</span>
<span class="attribution"><span class="source">Image from shutterstock.com</span></span>
</figcaption>
</figure>
<p>Resident <a href="https://theconversation.com/explainer-what-are-stem-cells-14391">stem cells</a> are specialised cells found at various locations within the body. When activated, these cells multiply and then transform into other cell types required to replace worn out cells under conditions of normal tissue maintenance. </p>
<p>This work builds on <a href="http://www.ncbi.nlm.nih.gov/pubmed/22143790">previous studies</a> identifying the stem cell population in the nail bed by unveiling a signalling mechanism that could be exploited to enhance the amount of tissue that could be regrown. The potential for repair after injury appears very limited in many tissues and organs. Understanding how to enhance stem cell activation in these tissues may stimulate repair not previously thought possible.</p>
<p>The ability to switch on and mobilise resident stem cells in regeneration will be important in a wide range of new therapies, particularity for organs affected by injury or disease. On a world stage, momentum is currently growing for these types of strategies. It is clear that once refined, these approaches are sure to have a profound influence on many different aspects of clinical medicine, opening up the possibility of replacing diseased or injured tissues.</p>
<p>We may be some way off from the dream of replacing whole limbs in humans but recent progress confirms that by deepening our understanding of stem cell activation, we can directly unlock more regeneration in mammals than normally possible.</p><img src="https://counter.theconversation.com/content/15177/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>James Godwin works for Monash University - The Australian Regenerative Medicine Institute. He receives funding from the National Heart Foundation of Australia. He is affiliated with The London Regenerative Medicine Network.</span></em></p>Damage to vital organs, the spinal cord, or limbs can have an enormous impact on our ability to move, function – and even live. But imagine if you could restore these tissues back to their original condition…James Godwin, Research Fellow, Monash UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/143912013-05-17T03:57:28Z2013-05-17T03:57:28ZExplainer: what are stem cells?<figure><img src="https://images.theconversation.com/files/23994/original/b7zcpthv-1368752267.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">We have a lot of cells, but where did they arise from?</span> <span class="attribution"><span class="source">j.reed</span></span></figcaption></figure><p>In a paper <a href="http://www.sciencedirect.com/science/article/pii/S0092867413005710">published in Cell</a> yesterday, scientists from the US and Thailand have, for the first time, successfully produced embryonic stem cells from human skin cells.</p>
<p>That sounds interesting, but what are stem cells and where do they come from? </p>
<p>If you take a limb from a rose tree, and put it in soil, it will grow into a thriving bush. </p>
<p>But you might say: “Plants are special. This won’t work with animals.” Or will it? If you cut off a lizard’s tail, a <a href="http://onlinelibrary.wiley.com/doi/10.1002/jez.a.346/abstract">new tail may grow</a>. A lobster can <a href="http://www.gma.org/lobsters/allaboutlobsters/parts.html">grow back a lost claw</a>. </p>
<p>There is a <a href="http://www.dailymail.co.uk/sciencetech/article-2107236/You-CAN-live-forever--long-flatworm-say-scientists.html">special type of flatworm</a> that can be cut in half, again and again hundreds of times, and each half grows back into a full worm. </p>
<p>Similarly, if you cut out half a human liver, <a href="http://www.vivo.colostate.edu/hbooks/pathphys/digestion/liver/regen.html">it will grow back</a>. The story of <a href="http://en.wikipedia.org/wiki/Prometheus">Prometheus</a>, whose liver was eaten away by eagles and regrew each day, suggests that the Greeks of ancient times knew about regeneration of organs. </p>
<figure class="align-center zoomable">
<a href="https://images.theconversation.com/files/23995/original/vxz5xrsh-1368752359.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/23995/original/vxz5xrsh-1368752359.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/23995/original/vxz5xrsh-1368752359.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=421&fit=crop&dpr=1 600w, https://images.theconversation.com/files/23995/original/vxz5xrsh-1368752359.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=421&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/23995/original/vxz5xrsh-1368752359.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=421&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/23995/original/vxz5xrsh-1368752359.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=529&fit=crop&dpr=1 754w, https://images.theconversation.com/files/23995/original/vxz5xrsh-1368752359.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=529&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/23995/original/vxz5xrsh-1368752359.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=529&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
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<span class="caption"></span>
<span class="attribution"><span class="source">Wikimedia Commons</span></span>
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<p>This sort of regeneration is attributed to special cells called “<a href="http://learn.genetics.utah.edu/content/tech/stemcells/scintro/">stem cells</a>”. </p>
<h2>Reprogramming the workers</h2>
<p>Most of our cells are like many professional workers - they are hardened in their ways and can’t manage career changes. </p>
<p>Blood cells carry oxygen or fight disease, muscle cells expand and contract to move us around, nerve cells carry signals, skin cells form a protective layer over our bodies, and structures made up of kidney cells filter our blood. </p>
<p>The cells of most organs or tissues are referred to as “terminally differentiated” cells. They have specialised, and many won’t divide again. If they are damaged or die they will disappear. This is very important. </p>
<figure class="align-right zoomable">
<a href="https://images.theconversation.com/files/23996/original/b4rr6mmy-1368752508.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/23996/original/b4rr6mmy-1368752508.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/23996/original/b4rr6mmy-1368752508.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=450&fit=crop&dpr=1 600w, https://images.theconversation.com/files/23996/original/b4rr6mmy-1368752508.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=450&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/23996/original/b4rr6mmy-1368752508.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=450&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/23996/original/b4rr6mmy-1368752508.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=566&fit=crop&dpr=1 754w, https://images.theconversation.com/files/23996/original/b4rr6mmy-1368752508.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=566&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/23996/original/b4rr6mmy-1368752508.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=566&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
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<span class="attribution"><span class="source">xopherlance</span></span>
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<p>Although we feel like we grow a lot after we are born, we really only double in size two or three times and most of our cells don’t divide much. </p>
<p>If they did we would be at great risk from <a href="https://theconversation.com/explainer-what-is-cancer-1673">cancer</a> – the uncontrolled doubling of cells at the wrong time. </p>
<p>We have a lot of cells and it is important that none of them run out of control. </p>
<p>But some cells can double to renew themselves and can also differentiate and give rise to specialised progeny. </p>
<p>These are the stem cells. We need them to produce new skin to replace damaged skin cells. Similarly, we need them in our guts to replace damaged cells on the surface of our intestines. </p>
<p>Our blood cells also get worn out as they race around our bodies so we have blood stem cells that divide and replace themselves. They also differentiate to form the different types of white and red blood cells we need. </p>
<p>Australian researchers <a href="http://cancerres.aacrjournals.org/content/66/20/9798.short">identified stem cells in the breast</a> that can proliferate and form a complete functioning breast. There are also stem cells in the brain and in the heart. </p>
<p>While stem cells tend to be very rare, they exist in many of our organs. </p>
<h2>Types of stem cells</h2>
<p>The ultimate stem cells are embryonic stem cells. </p>
<p>These cells are found in the inner cell mass of the early embryo and are referred to as “<a href="http://www.explorestemcells.co.uk/totipotentstemcells.html">totipotent</a>” since they have the ability to form every cell that is needed in the growing embryo. </p>
<p>They can be extracted from the early embryo and grown in culture dishes. </p>
<figure class="align-left zoomable">
<a href="https://images.theconversation.com/files/23997/original/y6fjsj7v-1368752659.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/23997/original/y6fjsj7v-1368752659.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/23997/original/y6fjsj7v-1368752659.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=900&fit=crop&dpr=1 600w, https://images.theconversation.com/files/23997/original/y6fjsj7v-1368752659.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=900&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/23997/original/y6fjsj7v-1368752659.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=900&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/23997/original/y6fjsj7v-1368752659.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=1131&fit=crop&dpr=1 754w, https://images.theconversation.com/files/23997/original/y6fjsj7v-1368752659.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=1131&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/23997/original/y6fjsj7v-1368752659.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=1131&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
<figcaption>
<span class="caption"></span>
<span class="attribution"><span class="source">euthman</span></span>
</figcaption>
</figure>
<p>They can also be genetically modified by the addition of DNA, then injected back into other embryos or into adult animals where find their way into localities that suit them and replace themselves by duplication or differentiate into other cell types that may be needed. For a long time this type of work had been done primarily in laboratory mice.</p>
<p>The techniques in yesterday’s Cell paper involved injecting the nucleus from a human skin cell into a human egg (the nucleus of which has been destroyed) then growing the resulting embryo until the inner cell mass cells could be harvested. </p>
<p>The method may still be controversial because it uses unfertilised eggs, but many people will regard it as preferable to using human embryos. And there are other interesting methods for making stem cells. </p>
<h2>Somatic cells to stem cells</h2>
<p>It is also possible to convert skin cells, and indeed many different terminally differentiated cells, back into what are called “<a href="http://en.wikipedia.org/wiki/Induced_pluripotent_stem_cell">induced pluripotent stem cells</a>” or iPS cells. </p>
<p>One uses the “magic four” or “OKSM” set of DNA-binding proteins that govern normal stem cell biology:</p>
<ul>
<li>Octamer-binding transcription factor 4 (<a href="http://en.wikipedia.org/wiki/Oct-4">OCT4</a>)</li>
<li>Kruppel-like factor 4 (<a href="http://en.wikipedia.org/wiki/KLF4">KLF4</a>)</li>
<li>SRY (sex determining region Y)-box 2 (<a href="http://en.wikipedia.org/wiki/SOX2">SOX2</a>)</li>
<li>cellular myelocytomatosis virus-like gene (<a href="http://en.wikipedia.org/wiki/Myc">MYC</a>)</li>
</ul>
<p>In 2012 <a href="http://www.nobelprize.org/nobel_prizes/medicine/laureates/2012/yamanaka.html">Shinya Yamanaka</a> won the Nobel Prize for discovering how to convert normal cells into iPS cells using the OKSM regulators to turn on and off the right genes and convert skin cells into stem cells. </p>
<p>Researchers are <a href="http://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0045603">continuing to investigate</a> whether iPS cells have the same therapeutic potential as embryo derived stem cells. </p>
<p>It is hoped that stem cells may provide therapies for people suffering from degenerative diseases. </p>
<p>Skin cells could be taken from a patient, converted to stem cells, and then these could be injected back into the damaged organ. </p>
<p>Ideally, they would repopulate the damaged organ with new cells.</p>
<p>So why doesn’t this happen in normal biology? Why aren’t our own heart stem cells busy trying to repair broken hearts? </p>
<p>They may be but our natural supply of stem cells is limited and presumably insufficient to tackle severe disease. </p>
<p>So why don’t we just have more stem cells in our bodies? </p>
<figure class="align-right zoomable">
<a href="https://images.theconversation.com/files/23998/original/7swfjk9q-1368752715.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/23998/original/7swfjk9q-1368752715.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/23998/original/7swfjk9q-1368752715.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=502&fit=crop&dpr=1 600w, https://images.theconversation.com/files/23998/original/7swfjk9q-1368752715.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=502&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/23998/original/7swfjk9q-1368752715.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=502&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/23998/original/7swfjk9q-1368752715.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=630&fit=crop&dpr=1 754w, https://images.theconversation.com/files/23998/original/7swfjk9q-1368752715.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=630&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/23998/original/7swfjk9q-1368752715.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=630&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
<figcaption>
<span class="caption">Lung cancer cells.</span>
<span class="attribution"><span class="source">Wellcome Images</span></span>
</figcaption>
</figure>
<p>The down side of having too many stem cells may be cancer. </p>
<p>Stem cells share a number of features with cancer cells – both are able to self-renew and double without limit. </p>
<p>One <a href="http://www.nature.com/labinvest/journal/v86/n12/full/3700488a.html">theory about cancer</a> holds that the disease most often originates not from terminally differentiated cells but from one of the small number of stem cells in the relevant tissues. </p>
<p>The obvious concern about using stem cells for therapy is that injecting too many could increase the chances that some of these cells would proliferate beyond control, and ultimately give rise to cancer. </p>
<p>Stem cell therapy for regenerative medicine is an exciting idea. </p>
<p>Every day we are learning more about stem cells - how to purify or make them, and how to grow them in culture and direct them down particular pathways to repopulate different organs. </p>
<p>Future research will assess the risks and how effective they can be in experimental systems and ultimately in human patients.</p><img src="https://counter.theconversation.com/content/14391/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Merlin Crossley receives funding from the University of New South Wales, the Australian Research Council and the National Health and Medical Research Council.</span></em></p>In a paper published in Cell yesterday, scientists from the US and Thailand have, for the first time, successfully produced embryonic stem cells from human skin cells. That sounds interesting, but what…Merlin Crossley, Dean of Science and Professor of Molecular Biology, UNSW SydneyLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/100492012-10-10T00:53:51Z2012-10-10T00:53:51ZNobel prize winners prove that success can be cloned<figure><img src="https://images.theconversation.com/files/16349/original/jxhgbzch-1349826420.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Shinya Yamanaka and John Gurdon have received the 2012 Nobel prize for Physiology or Medicine.</span> <span class="attribution"><span class="source">AAP</span></span></figcaption></figure><p>The 2012 <a href="http://www.nobelprize.org/nobel_prizes/medicine/laureates/2012/#">Nobel Prize for Medicine and Physiology</a> has been awarded to John Gurdon and Shinya Yamanaka, “for the discovery that mature cells can be reprogrammed to become pluripotent”.</p>
<p>A pluripotent cell is capable of becoming any of the cells in the body, just as the cells of an early embryo can. When they are derived from an embryo, these cells are referred to as embryonic stem cells and they have the potential to differentiate into all cell types of the body.</p>
<p>John Gurdon’s contribution to this field was his foresight, in 1958, to predict that the egg, which is normally fertilised to produce an embryo, contains powerful factors that regulate the way genes are expressed during very early development. </p>
<p>Gurdon used nuclei from mature frog cells to show that these nuclei were capable of being reprogrammed to a “naïve” state by frog eggs – so much so that they gave rise to a live offspring. What had actually happened was that the egg managed to wipe off specific marks present in the cell’s nuclei that enable them to perform specific functions, making the cell pluripotent and able to differentiate into all cell types of the body.</p>
<p>Gurdon’s work opened up a whole new field in developmental biology, which we call <a href="https://theconversation.com/think-you-can-think-yourself-better-think-again-558">epigenetics</a>. This involves the study of how the expression of genes is regulated. Some epigenetic factors silence genes so that they are not expressed, while others promote the expression of genes. </p>
<p>It enabled us to start understanding the importance of the timing of the expression of certain genes during development, and the whole sequence of gene expression, which determines how we develop. We now apply this knowledge to differentiating embryonic and induced pluripotent stem cells to understand when a mutation might have its first effect on an individual during development, and how it might predispose people to certain diseases.</p>
<figure class="align-center ">
<img alt="" src="https://images.theconversation.com/files/16303/original/hwyv5jf4-1349746237.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/16303/original/hwyv5jf4-1349746237.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=401&fit=crop&dpr=1 600w, https://images.theconversation.com/files/16303/original/hwyv5jf4-1349746237.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=401&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/16303/original/hwyv5jf4-1349746237.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=401&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/16303/original/hwyv5jf4-1349746237.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=504&fit=crop&dpr=1 754w, https://images.theconversation.com/files/16303/original/hwyv5jf4-1349746237.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=504&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/16303/original/hwyv5jf4-1349746237.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=504&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">Dolly the Sheep became a worldwide sensation and popularised the term ‘cloning’.</span>
<span class="attribution"><span class="source">Toni Barros/Wikimedia Commons</span></span>
</figcaption>
</figure>
<p>But it wasn’t until 1997 that one of the major advances resulted from Gurdon’s work. This was the cloning of Dolly the Sheep. Here, Keith Campbell and Ian Wilmut took a cell from a sheep mammary gland (breast tissue) and introduced the cell into a sheep egg. This resulted in Dolly, the first mammalian clone. Since then, multiple species have been cloned and the cloning industry has expanded to produce specialised livestock for agricultural purposes.</p>
<p>Scientists also wanted to know whether it was possible to derive human embryonic stem cells using this approach, where the cell to be reprogrammed would, for example, contain mutations associated with specific human diseases. This was considered to be a holy grail for scientists as it would provide human models of disease that are so desperately required for medical research. Sadly, this has proven to be elusive to date.</p>
<p>Although this is exciting work, cloning opened up a whole world of controversy. Research governance bodies such as the <a href="http://www.hfea.gov.uk/">Human Fertilisation and Embryology Authority</a> in the United Kingdom and the <a href="http://www.nhmrc.gov.au/">NHMRC</a> in Australia had to rethink whether they should allow people to do this sort of work using human cells and human eggs.</p>
<p>Nevertheless, a major breakthrough came when Shinya Yamanaka deciphered which of the key factors present in the egg conferred pluripotency. He chose four genes that, when packaged appropriately and then incubated with cells, such as skin cells, had the potential to reprogram or de-differentiate these cells into pluripotent stem cells. Remarkably, this technique did not require the use of an egg to generate these embryonic-like stem cells and became known as induced pluripotency.</p>
<p>Yamanaka then showed that these cells could be differentiated into many of the cell types of the body, such as nerve and heart cells. Since then, others have made induced pluripotent stem cell lines of various diseases including Parkinson’s disease.</p>
<p>The modification of DNA and how genes are expressed is not just limited to developmental biology. These scientists have allowed us to think differently about how, for example, cancers arise. Some of the factors involved in inducing pluripotency are similar to the factors involved in cancers. And this work has been instrumental for getting researchers from different disciplines to work together to find common ground and answer significant scientific questions.</p>
<figure class="align-center ">
<img alt="" src="https://images.theconversation.com/files/16301/original/s9tqrn22-1349745726.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/16301/original/s9tqrn22-1349745726.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=447&fit=crop&dpr=1 600w, https://images.theconversation.com/files/16301/original/s9tqrn22-1349745726.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=447&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/16301/original/s9tqrn22-1349745726.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=447&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/16301/original/s9tqrn22-1349745726.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=561&fit=crop&dpr=1 754w, https://images.theconversation.com/files/16301/original/s9tqrn22-1349745726.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=561&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/16301/original/s9tqrn22-1349745726.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=561&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">Stem cells derived from human embryonic cells can be manipulated to create other cells.</span>
<span class="attribution"><span class="source">Nissim Benvenisty/Wikimedia Commons</span></span>
</figcaption>
</figure>
<p>This is not the first Nobel prize associated stem cell research either. Martin J. Evans, Mario Capecchi and Oliver Smithies were <a href="http://www.nobelprize.org/nobel_prizes/medicine/laureates/2007/">awarded</a> the prize in 2007, “for the discoveries of principles for introducing specific gene modifications in mice by the use of embryonic stem cells.” Their work led to more efficient mechanisms for generating transgenic mice that may be deficient of a specific gene or have one that is over-expressed. Such mice are invaluable in medical research as they are instrumental in studying disease and for drug discovery.</p>
<p>There’s still the potential for stem cells to be used for therapeutic purposes, but they are equally important for helping us understand developmental biology processes and for designing therapeutics based on what we learn from them.</p>
<p>But this award shows how basic science is fundamentally important for opening the doors of translational research. As we drive towards translational research, we need to remember that early fundamental science is integral and we can’t do translational work without it.</p>
<p>Science also needs young intellects to step forward to drive tomorrow’s research programs. Giving people like John Gurdon and Shinya Yamanaka the <a href="http://www.nobelprize.org/nobel_prizes/medicine/laureates/2012/index.html?pageNum_GetGreetings=2&totalRows_GetGreetings=1093">Nobel prize</a> for their inventiveness will hopefully switch young people on and encourage them to think into the unknown. It’s also worth remembering that John Gurdon was told by his teacher that he was not cut out for science.</p><img src="https://counter.theconversation.com/content/10049/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Justin St. John holds a patent on cloning. He receives funding from the NHMRC and Australian Pork Limited, and received funding from the MRC in the UK. He reviews license applications for the Human Fertilisation and Embryology Authority in the UK and held a license to make human stem cells using cross-species cloning technology.</span></em></p>The 2012 Nobel Prize for Medicine and Physiology has been awarded to John Gurdon and Shinya Yamanaka, “for the discovery that mature cells can be reprogrammed to become pluripotent”. A pluripotent cell…Justin St. John, Professor and Director, Centre for Genetic Diseases, Monash Institute of Medical Research, Monash UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/25242012-02-06T10:18:04Z2012-02-06T10:18:04ZSafety in numbers: how three parents can beat genetic diseases<figure><img src="https://images.theconversation.com/files/7417/original/m2trh3nf-1328522089.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">The mitochondrial genome is passed on by mothers – defects and all.</span> <span class="attribution"><span class="source">Marcos Leal</span></span></figcaption></figure><p><strong>Media outlets have been reporting that scientists are planning to “create designer babies” with three parents. Professor Justin St John, Director of the Centre for Reproduction and Development at Monash University explains the technology being proposed and its feasibility.</strong></p>
<p>We inherit most genes from both parents, which determine things like hair colour, eye colour and behavioural patterns, but there’s a small proportion of DNA that we inherit only from our mothers. This DNA is from a region of the ovum (egg) called the cytoplasm, which houses organelles called mitochondria. </p>
<p>One of the main functions of mitochondria is to generate energy and that’s why they’re known as the powerhouses of the cell. Within each mitochondria, there’s a small round piece of DNA called the mitochondrial genome (mtDNA). This has some very important genes because they’re directly involved in the process of making energy. If any of these genes are mutated, then the individual will have severe disabilities related to not being able to generate enough energy or some of their cells will not function properly. </p>
<p>So we inherit our mtDNA only from our mother and what we inherit is the mtDNA that’s in her eggs just prior to fertilisation. Then our father’s sperm comes along and it delivers the chromosomal genes which join with our mother’s chromosomal genes and that’s the bulk of the DNA in the body. </p>
<p>The content of mtDNA gets changed during various stages of the development of the embryo and the foetus, while chromosomal DNA comes together and is then replicated or copied and will appear in each new cell. When the embryo starts to form all the different tissues of the body, the mutant mtDNA can go to some of the cells of the body – it can be distributed quite randomly. </p>
<p>So we can never predict if mutant mtDNA is going to distribute to cells which give rise to the brain, the heart, the kidneys or whatever. What happens is that if the mutant molecules go to cells that have a high requirement for energy, such as the brain or the muscle cells, then you’re likely to be hit by mitochondrial disease. If it distributes to tissues that are not necessarily high-energy requiring cells, then the likelihood of being affected is far less.</p>
<p>Currently, there are technologies that work with embryos, such as pre-implantation genetic diagnosis, which is used in IVF clinics. When an embryo forms, it starts by having two cells and then four, eight, sixteen onwards. This technology takes one of these cells and analyses it to see whether that embryo (and the child that it will form into), will be affected by the specific genetic disease.</p>
<p>And that’s perfectly fine for diseases associated with the chromosomal genome. But because we don’t understand how the mitochondrial genome distributes early on during development, the information that we get from pre-implantation genetic diagnosis doesn’t usually help in avoiding mitochondrial diseases.</p>
<p>The more appropriate process (since we know the mtDNA is in the woman’s egg) is to take out the genome from that egg and transfer it into an egg of a woman who isn’t a carrier of mitochondrial disease. Then you can fertilise this newly created or reconstructed egg with the father’s sperm. So the new baby will have chromosomes from it’s mother and father but it will have another population of mtDNA, which will be from the woman who donated her egg to this treatment. That’s why people will refer to children who would result from this process as having three parents.</p>
<figure class="align-center ">
<img alt="" src="https://images.theconversation.com/files/7402/original/rf87hwf3-1328504034.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/7402/original/rf87hwf3-1328504034.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=400&fit=crop&dpr=1 600w, https://images.theconversation.com/files/7402/original/rf87hwf3-1328504034.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=400&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/7402/original/rf87hwf3-1328504034.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=400&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/7402/original/rf87hwf3-1328504034.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=503&fit=crop&dpr=1 754w, https://images.theconversation.com/files/7402/original/rf87hwf3-1328504034.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=503&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/7402/original/rf87hwf3-1328504034.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=503&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">The process of ensuring an embryo doesn’t carry mitochondrial disease.</span>
<span class="attribution"><span class="source">Justin St John</span></span>
</figcaption>
</figure>
<p>And we don’t know whether these children will resemble the third parent. This technology is very similar to the way we do cloning and it’s interesting to consider some of the studies that have used cloning technology to, for instance, make different types of fish. Researchers took a cell from a carp and they transferred into a goldfish egg. The resulting fish did have some of the characteristics of the egg donor. </p>
<p>There’s another approach to circumventing mitochondrial disease. Instead of taking an unfertilised egg, you start with one that’s already been fertilised (zygote), so it’s got two populations of chromosomal DNA. You can go in and take out both these populations and transfer them into another zygote that has had these two bits removed.</p>
<figure class="align-center ">
<img alt="" src="https://images.theconversation.com/files/7401/original/pggtchtm-1328504032.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/7401/original/pggtchtm-1328504032.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=400&fit=crop&dpr=1 600w, https://images.theconversation.com/files/7401/original/pggtchtm-1328504032.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=400&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/7401/original/pggtchtm-1328504032.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=400&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/7401/original/pggtchtm-1328504032.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=503&fit=crop&dpr=1 754w, https://images.theconversation.com/files/7401/original/pggtchtm-1328504032.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=503&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/7401/original/pggtchtm-1328504032.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=503&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">Another way to avoid mitochondrial disease.</span>
<span class="attribution"><span class="source">Justin St John</span></span>
</figcaption>
</figure>
<p>The problem with these processes is that we’re not sure that when you perform these transfers, you’re not going to take a few of the mutant mtDNA with you. We know that mtDNA distributes or segregates unequally during early development and we don’t know if it’s being selected for or selected against – whether it persists or is eliminated. And we don’t know that if you carry over a few of these mtDNA with mutations, whether that will actually still lead to the onset of mitochondrial disease. </p>
<p>So we need some rigorous experiments to determine whether it’s possible to extract the chromosomes either from the mum’s egg or the newly fertilised egg, without taking any mutant mtDNA along.</p>
<p><a href="http://www.ncbi.nlm.nih.gov/pubmed/19710649?dopt=Abstract&holding=npg">Authors of some studies</a> performed on monkeys in the United States have argued they didn’t carry mtDNA from the original egg when they performed this first process. But <a href="http://www.nature.com/gt/journal/v17/n2/full/gt2009164a.html">we suspect</a> their detection processes weren’t rigorous enough, and they didn’t look at every tissue in the monkeys they created. Because mtDNA distributes unequally during early development, you’d have to check all the tissues to be sure there are no passed on mutations.</p>
<p>In Australia, the <a href="https://legislationreview.nhmrc.gov.au/2010-legislation-review-committee">Heerey Committee</a> sat last year to review whether we should keep certain technologies going for experimental purposes. Whether, for instance, we should still use cloning to try and make embryonic stem cells with human eggs and human embryos. </p>
<p>One of the matters the Committee discussed in <a href="https://legislationreview.nhmrc.gov.au/sites/default/files/legislation_review_reports.pdf">their report</a> was whether we could try to make embryos by transferring the mother’s and father’s chromosomes from one egg to another under experimental conditions. And our interpretation is that it might be allowable under license from the NHMRC but an application would have to be made that would be ruled on.</p>
<p>The other process of taking the chromosomes from an unfertilised egg wouldn’t be allowed because we’re talking about generating a new embryo for research purposes. That’s where the distinction between the two processes lies.</p>
<p>What we need to do is get a large body of data that’s based on sound experimental analyses together. Then scientists would be in a strong position to go to the government and show these technologies have been validated. And the government could make an informed decision about whether we can travel down this path to eradicating mitochondrial disease.</p>
<p>There are going to be groups that won’t want this to happen. It’s quite a controversial technology because we’re dealing with embryos; some people accept this but others find it abhorrent. Nevertheless, we have the opportunity to prevent the next generation of children from having mitochondrial disease. </p><img src="https://counter.theconversation.com/content/2524/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Justin St. John receives funding from the NMHRC and used to receive funding from the MRC in the United Kingdom.</span></em></p>Media outlets have been reporting that scientists are planning to “create designer babies” with three parents. Professor Justin St John, Director of the Centre for Reproduction and Development at Monash…Justin St. John, Professor and Director, Centre for Genetic Diseases, Monash Institute of Medical Research, Monash UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/50192012-02-03T03:15:13Z2012-02-03T03:15:13ZSaving the snow leopard: stem-cell generation a bright new hope<figure><img src="https://images.theconversation.com/files/7344/original/kwrw4ff6-1328155999.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Researchers have taken important steps in conserving endangered cats.</span> <span class="attribution"><span class="source">dragaroo/Flickr</span></span></figcaption></figure><p>Looking at embryonic cells allows researchers to understand many of the fundamental questions about how an animal’s genes are structured and the role they play in developing the adult animal. This information is vital for conservation. But when the animal you’re working on is a snow leopard, harvesting these cells can be incredibly difficult.</p>
<p>Previously the only way to get these cells would be to take embryonic stem cells from snow leopard embryos. But, gametes (or sperm and eggs) from endangered animals are usually difficult to obtain, either due to feasibility, infertility issues, old age or because the animals have died.</p>
<p>Because of these difficulties, we have been generating stem-like cells - “induced Pluripotent Stem Cells” (iPSC) - from the cells of adult cats. Not only can these cells answer questions about animal development; they may help us to conserve endangered species.</p>
<p>In mice and pigs, iPSC technology can create all cell types of the individual, including the gametes, and can be generated from virtually any tissue source - even dead animals. </p>
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<p>For endangered species this would prove invaluable. The genetic pool of many endangered species is limited, and this leads to inbreeding depression and associated issues.</p>
<p>To generate the iPSC for our research we took <a href="http://en.wikipedia.org/wiki/Fibroblast">skin fibroblasts</a> - a type of cell - from a snow leopard named Mangal. Mangal had cancer and was being euthanized. </p>
<p>When generating mice or human iPSC, researchers routinely use four stem cell genes. We required an additional stem cell gene, called “Nanog”, to generate iPSC from a snow leopard. When we forced these five genes to express, it induced the adult skin cells to “reprogram” into embryonic stem-like cells (iPSC). We analysed the iPSC cells to show that we had reprogrammed them successfully and they could mature into multiple cell types.</p>
<p>These cells allow us to understand basic developmental biology in snow leopards. With refinement we believe iPSC will provide donor cells for efficient nuclear transfer (also called cloning).</p>
<p>Such cells have been coaxed into gametes, especially spermatozoa-like cells, in mice. Translation to snow leopard would provide a unique source of gametes for this endangered species, especially as (as we have shown) the cells can be obtained from an adult.</p>
<figure class="align-center ">
<img alt="" src="https://images.theconversation.com/files/7345/original/2qbtxb77-1328155999.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/7345/original/2qbtxb77-1328155999.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=480&fit=crop&dpr=1 600w, https://images.theconversation.com/files/7345/original/2qbtxb77-1328155999.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=480&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/7345/original/2qbtxb77-1328155999.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=480&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/7345/original/2qbtxb77-1328155999.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=603&fit=crop&dpr=1 754w, https://images.theconversation.com/files/7345/original/2qbtxb77-1328155999.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=603&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/7345/original/2qbtxb77-1328155999.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=603&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
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<span class="attribution"><span class="source">Timmy Toucan</span></span>
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<p>In mice, iPSC have successfully (albeit inefficiently) been combined with a host embryo to create a complete individual. Called embryo complementation, the process can be manipulated to allow the iPSC to give rise to a complete individual.</p>
<p>Translating this approach to endangered species would constitute a huge breakthrough for conservation. Again this is a long-term goal of ours.</p>
<p>Obviously all research, and especially research on exotic species, takes a considerable amount of funding and motivation, so our progress will largely be influenced by funding constraints. However we do have a long-term strategy which we will pursue as funds and personnel permit.</p>
<p>Extending this initial breakthrough to the generation of whole animals would provide a novel approach to conservation.</p><img src="https://counter.theconversation.com/content/5019/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Rajneesh Verma is a PhD student at Monash Institute of Medical Research, Monash university, melbourne.</span></em></p><p class="fine-print"><em><span>Paul Verma is an Adjunct Professor and Head of the Cell Reprogramming and Stem Cells Group at the Monash Institute of Medical Research at Monash University.</span></em></p>Looking at embryonic cells allows researchers to understand many of the fundamental questions about how an animal’s genes are structured and the role they play in developing the adult animal. This information…Rajneesh Verma, PhD Scholar, Monash UniversityPaul Verma, Laboratory Head, Monash Institute for Medical Research, Monash UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/42912011-12-13T19:43:38Z2011-12-13T19:43:38ZXenotransplantation: using pigs as organ and tissue donors for humans<figure><img src="https://images.theconversation.com/files/6337/original/tchnchvm-1323737775.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Pigs may be the answer to Australia's organ donor shortage.</span> <span class="attribution"><span class="source">Thornypup</span></span></figcaption></figure><p>Transplantation is the best available treatment for many serious health problems including diabetes, kidney failure and heart disease. These conditions affect millions of people worldwide and the cost of treatment, loss of productivity and reduced quality of life are enormously expensive to society. </p>
<p>Although transplantation offers a lifeline to these patients, there is far greater demand for organs and tissues than can ever be met using human donors. Even with the <a href="http://www.donatelife.gov.au/">government-driven push</a> to increase the donation rate in Australia, many patients will become too sick to receive a transplant or will die while on the waiting list. </p>
<p>Some scientists believe that <a href="https://theconversation.com/its-a-vision-thing-the-case-for-a-far-sighted-approach-to-stem-cell-research-1790">stem cells will ultimately provide a solution</a> to this pressing medical problem, but growing a highly complex organ from stem cells remains in the realms of science fiction, at least for now. </p>
<p>A treatment that is much closer to reality, and indeed has already entered early clinical trials, is the transplantation of animal organs, tissues or cells into humans. This is called xenotransplantation.</p>
<h2>Which species?</h2>
<p>Humans are primates, so the obvious choice of donor animal for xenotransplantation would appear to be another member of the primate family (chimpanzees and baboons, for instance) because of their physiological similarity. But non-human primates have been ruled out as donors for several compelling practical and ethical reasons. </p>
<p>One of the risks to transplant recipients is infection by viruses transmitted by the transplanted organ. As our closest cousins in the animal kingdom, primates are more likely than other animals to carry viruses capable of infecting humans; HIV, the virus responsible for AIDS, originated in chimpanzees. </p>
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<img alt="" src="https://images.theconversation.com/files/6341/original/zwtq9dgn-1323738604.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/6341/original/zwtq9dgn-1323738604.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=399&fit=crop&dpr=1 600w, https://images.theconversation.com/files/6341/original/zwtq9dgn-1323738604.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=399&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/6341/original/zwtq9dgn-1323738604.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=399&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/6341/original/zwtq9dgn-1323738604.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=502&fit=crop&dpr=1 754w, https://images.theconversation.com/files/6341/original/zwtq9dgn-1323738604.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=502&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/6341/original/zwtq9dgn-1323738604.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=502&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
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<span class="caption">Primates aren’t the best fit to donate organs and tissues to humans.</span>
<span class="attribution"><span class="source">Troy B Thompson</span></span>
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<p>This “relatedness” also poses ethical problems, with the public understandably reluctant to exploit animals that share many features with humans. And even if you discount the ethical question, it’s hard to imagine being able to breed enough primates to meet the increasing demand for donor organs.</p>
<p>Pigs, on the other hand, tick many of the boxes. They can be raised in a clean environment, so the risk of infection from pig donors may actually be lower than that from human donors. They are already widely bred for the food industry, solving the supply issue and, provided they are treated humanely, present less of an ethical dilemma. </p>
<p>Material from pigs has been routinely and safely used for medical purposes for decades, with <a href="http://www.theaustralian.com.au/news/nation/rudds-second-heart-valve-replacement-riskier/story-e6frg6nf-1226098607967">heart valves</a> the best known example. The evidence from animal models suggests that most pig organs will work properly in human recipients.</p>
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<img alt="" src="https://images.theconversation.com/files/6338/original/rw9m3cdb-1323738102.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/6338/original/rw9m3cdb-1323738102.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=450&fit=crop&dpr=1 600w, https://images.theconversation.com/files/6338/original/rw9m3cdb-1323738102.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=450&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/6338/original/rw9m3cdb-1323738102.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=450&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/6338/original/rw9m3cdb-1323738102.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=566&fit=crop&dpr=1 754w, https://images.theconversation.com/files/6338/original/rw9m3cdb-1323738102.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=566&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/6338/original/rw9m3cdb-1323738102.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=566&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
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<span class="caption">There are still some major barriers to overcome before xenotransplantation becomes a clinical possibility.</span>
<span class="attribution"><span class="source">Ro Irving</span></span>
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<p>On the downside, the evolutionary distance between pigs and humans means that the human immune system mounts a very strong response to pig organs. The drugs that are used to prevent rejection of human transplants are simply not powerful enough when it comes to pig transplants. </p>
<p>One solution for this problem is to genetically modify pigs so that their organs will not be recognised as foreign when transplanted into humans. <a href="http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(11)61091-X/abstract">Several groups</a> around the world, including in Australia, have produced GM pigs for xenotransplantation research. These pigs are still in the testing phase, but the progress that has been made over the last 10 years suggests that the move to the clinic is not too far away. </p>
<h2>Treating diabetes with pig islets</h2>
<p>Pigs may also be the key to future treatment of diabetes. Insulin, the hormone that controls the level of sugar in the blood, is made by clusters of cells in the pancreas called islets. People with type 1 diabetes have abnormally high blood sugar because their islets are destroyed by the immune system. While regular insulin injections restore some control, the long term prospects are poor, with complications including renal failure and blindness. </p>
<p>Transplantation with human islets is an option open to only a handful of patients. Pig islets are an attractive alternative, because pig insulin is 98% identical to human insulin and was used to treat patients before recombinant human insulin became available.</p>
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<img alt="" src="https://images.theconversation.com/files/6339/original/txhy9bkg-1323738187.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/6339/original/txhy9bkg-1323738187.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=600&fit=crop&dpr=1 600w, https://images.theconversation.com/files/6339/original/txhy9bkg-1323738187.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=600&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/6339/original/txhy9bkg-1323738187.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=600&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/6339/original/txhy9bkg-1323738187.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=754&fit=crop&dpr=1 754w, https://images.theconversation.com/files/6339/original/txhy9bkg-1323738187.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=754&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/6339/original/txhy9bkg-1323738187.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=754&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
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<span class="caption">Pig insulin was used for decades to treat people with diabetes.</span>
<span class="attribution"><span class="source">Jill A Brown</span></span>
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<p>In a clinical trial currently taking place in New Zealand, pig islets contained within microcapsules have been injected into the abdomen of 11 patients with diabetes. The microcapsules allow nutrients to get in and insulin to get out, but importantly they also protect the pig islets from the recipient’s immune system so that no anti-rejection drugs are needed. Early results suggest that the microcapsule treatment will not be a complete cure, but may benefit patients with severe diabetes. </p>
<p>In the meantime, many other strategies are being explored. <a href="http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(11)61091-X/abstract">Results from animal models</a> showing islets from GM pigs can reverse diabetes for many months are particularly encouraging. </p>
<h2>Future xenotransplantation</h2>
<p>A recent <a href="http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(11)61091-X/abstract">review in the prestigious medical journal The Lancet</a> is carefully optimistic that clinical xenotransplantation may soon become a reality, particularly for cellular grafts such as islets. Will this be, as suggested by the authors of the review, the “next medical revolution”? We’ll have to wait and see.</p><img src="https://counter.theconversation.com/content/4291/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Peter Cowan receives funding from the National Health and Medical Research Council of Australia (NHMRC) and the Juvenile Diabetes Research Foundation (JDRF).</span></em></p>Transplantation is the best available treatment for many serious health problems including diabetes, kidney failure and heart disease. These conditions affect millions of people worldwide and the cost…Peter Cowan, Co-director of the Immunology Research Centre, St Vincent's Hospital MelbourneLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/38982011-10-25T03:11:08Z2011-10-25T03:11:08ZNothing like mother’s milk: potential treatments from stem cells in breast milk<figure><img src="https://images.theconversation.com/files/4777/original/6132906504_0e14c84024_b.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">This study suggests breast milk is much more than nutrition for the baby, it could also play a role in stem cell therapies.</span> <span class="attribution"><span class="source">various brennemans/flickr</span></span></figcaption></figure><p>Researchers have identified stem cells in human breast milk, which <a href="http://www.news.uwa.edu.au/201110174047/awards-and-prizes/breastmilk-natural-stem-cell-therapy#pagecontainer">behave similarly to embryonic stem cells when cultivated</a> in a medium containing nutrients. </p>
<p>The finding suggests breast milk could be used as a non-invasive and plentiful source of stem cells, which also bypass the ethical concerns surrounding the use of embryonic stem cells, for innovative stem cell therapies. </p>
<p>Stem cell therapy is a promising and rapidly developing field, as it could enable treatment of currently fatal diseases. One of the limiting factors to developing successful therapies is the source of stem cells. </p>
<p>There’s much debate about the ethics of using human embryonic stem cells, which have the potential to differentiate into most human adult cell types. </p>
<p>This ability to differentiate into almost all body cell types makes embryonic stem cells unique, and has not been found in adult stem cells, such as those from the bone marrow. </p>
<p>Stem cells found in breast milk have now been demonstrated to have many of the properties of embryonic stem cells, including the ability to regenerate, but also differentiate into many different body cell types.</p>
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<p>Under specific cultivating conditions that mimic the microenvironment of specific organs, breast milk stem cells can turn into various cell types of other organs and tissues, such as bone, cartilage, fat, liver and pancreas.</p>
<p>Based on these findings, breast milk stem cells offer an exciting new opportunity for an ethical, non-invasive and plentiful source of stem cells that can potentially be used in novel stem cell therapies for the benefit not only of the mother and the child, but also other people. </p>
<p>Since these cells display the potential to become insulin-producing pancreatic cells, the discovery could, for instance, open up new avenues for a stem cell-based therapy for diabetes. </p>
<p>And the ability of these cells to turn into nerve cells could allow further applications in treating neurodegenerative diseases, such as Parkinson’s disease.</p>
<p>The enormous potential for using the body’s own (autologous), but also other people’s cells (allogeneic) in stem cell therapies extends to the potential to treat fatal neonatal diseases using breast milk cells.</p>
<p>Breast milk stem cells can also be used as a physiological model to study the malignant transformation that occurs in breast cancer. </p>
<p>This could potentially assist the understanding of what leads to cancer, and how it can be successfully treated.</p>
<p>Finally, the finding also opens a new discussion about the role of these cells for the breast-fed infant.</p>
<p>Breast milk immune cells have been shown to pass through the infant’s gut into the blood circulation and engraft in various tissues. </p>
<p>We hypothesise that stem cells from breast milk are also able to do so, contributing to infant development early in life. </p>
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<p>These findings suggest that breast milk is much more than nutrition for the baby, and therefore it can never be replaced or substituted by infant formula.</p>
<p>Future research will examine the potential and properties of breast milk stem cells, including their function in the lactating breast; their normal function(s) in the breast-fed baby; their use as models in breast cancer research; and their potential for in vivo cell transplantation and stem cell therapies.</p>
<p>Breast milk stem cell research holds a lot of promise for our understanding of how the human body works and how disease occurs, as well as for the development of novel therapies for a range of medical conditions.</p><img src="https://counter.theconversation.com/content/3898/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Foteini Hassiotou receives funding from Medela AG, the Women and Infants Research Foundation of Western Australia, and the University of Western Australia.</span></em></p>Researchers have identified stem cells in human breast milk, which behave similarly to embryonic stem cells when cultivated in a medium containing nutrients. The finding suggests breast milk could be used…Foteini Hassiotou, Post-doctoral researcher in Stem Cell biology and Human Nutrition, The University of Western AustraliaLicensed as Creative Commons – attribution, no derivatives.