tag:theconversation.com,2011:/au/topics/leukaemia-3090/articlesLeukaemia – The Conversation2021-09-08T20:12:17Ztag:theconversation.com,2011:article/1660332021-09-08T20:12:17Z2021-09-08T20:12:17Z20 years on, 9/11 responders are still sick and dying<figure><img src="https://images.theconversation.com/files/419188/original/file-20210903-23797-akqghk.jpg?ixlib=rb-1.1.0&rect=0%2C0%2C2396%2C1595&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">
</span> <span class="attribution"><a class="source" href="https://photos.aap.com.au/search/world%20trade%20center%20attack">Shawn Baldwin/AP/AAP Image</a></span></figcaption></figure><p>Emergency workers and clean-up crew are among 9/11 responders still suffering significant health issues 20 years after the <a href="https://www.history.com/topics/21st-century/9-11-attacks">terrorist attacks</a>.</p>
<p>More than <a href="https://pubmed.ncbi.nlm.nih.gov/18500709/">91,000 workers and volunteers</a> <a href="https://www1.nyc.gov/site/911health/enrollees/rescue-recovery-workers.page">were exposed</a> to a range of hazards during the rescue, recovery and clean-up operations.</p>
<p>By March 2021, some 80,785 of these responders had enrolled in the <a href="https://www.cdc.gov/wtc/">World Trade Center Health Program</a>, which was set up after the attacks to monitor their health and treat them.</p>
<p>Now our <a href="https://www.cambridge.org/core/journals/prehospital-and-disaster-medicine/article/abs/health-trends-among-911-responders-from-20112021-a-review-of-world-trade-center-health-program-statistics/09B87521287B943402782DAADB47E0B9">published research</a>, which is based on examining these health records, shows the range of physical and mental health issues responders still face.</p>
<h2>Breathing problems, cancer, mental illness</h2>
<p>We found 45% of responders in the health program have aerodigestive illness (conditions that affect the airways and upper digestive tract). A total of 16% have cancer and another 16% have mental health illness. Just under 40% of responders with health issues are aged 45-64; 83% are male.</p>
<p>Our analysis shows 3,439 of responders in the health program are now dead — far more than the <a href="https://parade.com/1248604/jessicasager/9-11-facts/">412 first responders who died on the day</a> of the attacks.</p>
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<p>Respiratory and upper digestive tract disorders are the number one cause of death (34%), ahead of cancer (30%) and mental health issues (15%). </p>
<p>Deaths attributed to these three factors, as well as musculoskeletal and acute traumatic injuries, have increased six-fold since the start of 2016. </p>
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Read more:
<a href="https://theconversation.com/how-the-pain-of-9-11-still-stays-with-a-generation-64725">How the pain of 9/11 still stays with a generation</a>
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<h2>An ongoing battle</h2>
<p>The number of responders enrolling in the health program with emerging health issues rises each year. More than 16,000 responders have enrolled in the past five years. </p>
<p>Cancer is up 185% over the past five years, with leukaemia emerging as particularly common, overtaking colon and bladder cancer in the rankings.</p>
<p>This equates to an increase of 175% in leukaemia cases over a five-year period, which is not surprising. There is a <a href="https://pubmed.ncbi.nlm.nih.gov/32771228/">proven link</a> between benzene exposure and acute myeloid leukaemia. Benzene is found in jet fuel, one of the toxic exposures at the World Trade Center. And acute myeloid leukaemia is one of the main types of leukaemia reported not only by responders, but by <a href="https://www.wtc-illness.com/cancers/leukemia-blood-cancer">residents of lower Manhattan</a>, who also have higher-than-normal rates. </p>
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<p>Prostate cancer is also common, increasing 181% since 2016. Although this fits with the age profile of many of the health program’s participants, some responders are developing an <a href="https://pubmed.ncbi.nlm.nih.gov/31221798/">aggressive, fast-growing form</a> of prostate cancer. </p>
<p>Inhaling the toxic dust at the World Trade Center site may trigger a cascading series of cellular events, increasing the number of inflammatory T-cells (a type of immune cell) in some of the responders. This increased inflammation <a href="https://pubmed.ncbi.nlm.nih.gov/26816843/">may eventually lead to prostate cancer</a>.</p>
<p>There may also be a <a href="https://pubmed.ncbi.nlm.nih.gov/31490535/">significant link between</a> greater exposure at the World Trade Center and a higher risk of long-term cardiovascular disease (disease affecting the heart and blood vessels). Firefighters who responded to the World Trade Center on the morning of the attacks were 44% more likely to develop cardiovascular disease than those who arrived the next day.</p>
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Read more:
<a href="https://theconversation.com/air-pollution-causes-cancer-so-lets-do-something-about-it-19380">Air pollution causes cancer, so let's do something about it </a>
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<h2>The mental health effects</h2>
<p>About <a href="https://pubmed.ncbi.nlm.nih.gov/31625489/">15-20%</a> of responders are estimated to be living with <a href="https://www.beyondblue.org.au/the-facts/anxiety/types-of-anxiety/ptsd">post-traumatic stress disorder</a> (PTSD) symptoms — roughly <a href="https://www.nimh.nih.gov/health/statistics/post-traumatic-stress-disorder-ptsd">four times</a> the rate of the general population. </p>
<p>Despite 20 years having passed, PTSD <a href="https://pubmed.ncbi.nlm.nih.gov/28805168/">is a growing problem</a> for responders. Almost half of all responders <a href="https://pubmed.ncbi.nlm.nih.gov/31776767/">report</a> they need ongoing mental health care for a range of mental health issues including PTSD, anxiety, depression and <a href="https://www.medicalnewstoday.com/articles/325578">survivor guilt</a>.</p>
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Read more:
<a href="https://theconversation.com/9-11-anniversary-a-watershed-for-psychological-response-to-disasters-2975">9/11 anniversary: a watershed for psychological response to disasters</a>
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<p>Researchers <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7364857/">have also found</a> brain scans of some responders indicate the onset of early-stage dementia. This is consistent with <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7364857/">previous work</a> noting cognitive impairment among responders occurs at about twice the rate of people 10-20 years older.</p>
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<h2>COVID-19 and other emerging threats</h2>
<p>Responders’ underlying health conditions, such as cancer and respiratory ailments, have also left them <a href="https://www.usatoday.com/in-depth/news/nation/2021/05/05/covid-risk-911-september-2001-ground-zero-responders-causes-concern/4961779001/">vulnerable to COVID-19</a>. By the end of August 2020, <a href="https://www.newsweek.com/how-many-people-died-911-thousands-perishing-september-11-related-illnesses-1531058">some 1,172 responders</a> had confirmed COVID-19.</p>
<p>Even among responders who have not been infected, the pandemic <a href="https://www.thecity.nyc/2020/9/10/21431746/how-many-9-11-survivors-have-died-of-covid-19">has exacerbated</a> one of the key conditions caused by search and rescue, and recovery after terrorist attacks — PTSD.</p>
<p><a href="https://www.nbcnews.com/news/us-news/covid-19-has-killed-dozens-9-11-first-responders-n1239885">More than 100 responders have died</a> due to complications from the virus, which has also exacerbated other responders’ PTSD symptoms.</p>
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<p>The number of responders with cancers associated with asbestos exposure at the World Trade Center is expected to rise in coming years. This is because mesothelioma (a type of cancer caused by asbestos) usually takes <a href="https://www.canceraustralia.gov.au/cancer-types/mesothelioma-cancer/awareness#:%7E:text=It%20usually%20takes%20a%20very,and%20roofing%2C%20and%20in%20insulation.">20-50 years to develop</a>. </p>
<p>As of 2016, at least 352 responders had been diagnosed with the lung condition <a href="https://www.mayoclinic.org/diseases-conditions/asbestosis/symptoms-causes/syc-20354637">asbestosis</a>, and at least 444 had been diagnosed with another lung condition, <a href="https://www.mayoclinic.org/diseases-conditions/pulmonary-fibrosis/symptoms-causes/syc-20353690">pulmonary fibrosis</a>. Exposure to asbestos and other fibres in the toxic dust <a href="https://www.asbestos.com/world-trade-center/">may have contributed</a>.</p>
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Read more:
<a href="https://theconversation.com/health-harms-of-asbestos-wont-be-known-for-decades-14845">Health harms of asbestos won't be known for decades </a>
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<h2>Lessons learned</h2>
<p>Our research involved analysing data from existing databases. So we cannot make direct links between exposure at the World Trade Center site, length of time there, and the risk of illness. </p>
<p>Differences in age, sex, ethnicity, smoking status and other factors between responders and non-responders should also be considered. </p>
<p>Increased rates of some cancers in some responders may also be associated with <a href="https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2764101">heightened surveillance</a> rather than an increase in disease.</p>
<p>Nevertheless, we are now beginning to understand the long-term effects of responding to the 9/11 terrorist attacks. Exposure is still having both a physical and mental health impact and it’s likely responders are still developing illnesses related to their exposures.</p>
<p>Ongoing monitoring of responders’ health remains a priority, especially considering the looming threat of new asbestos-related cancers.</p><img src="https://counter.theconversation.com/content/166033/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>More 9/11 responders died from physical and mental health issues after the terrorist attacks than on the day itself. And survivors are still suffering 20 years later.Erin Smith, Associate Professor in Disaster and Emergency Response, School of Medical and Health Sciences, Edith Cowan UniversityBrigid Larkin, PhD candidate, Edith Cowan UniversityLisa Holmes, Lecturer, Paramedical Science, School of Medical and Health Sciences, Edith Cowan UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/1033292018-11-08T00:33:57Z2018-11-08T00:33:57ZWhat it means when scientists say their results are ‘significant’<figure><img src="https://images.theconversation.com/files/244035/original/file-20181106-74769-fmysng.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Sometimes statistical analysis suggests a result is significant – but actually in real life it means very little. </span> <span class="attribution"><a class="source" href="https://unsplash.com/photos/x5i1zcMlPEg">Marlon Lara/Unsplash</a>, <a class="license" href="http://creativecommons.org/licenses/by/4.0/">CC BY</a></span></figcaption></figure><p><em>This article is part of the series <strong><a href="https://theconversation.com/au/topics/this-is-research-61770">This is research</a></strong>, where we ask academics to share and discuss open access articles that reveal important aspects of science. Today’s piece looks at statistics, and how to interpret them for meaning in the real world.</em></p>
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<p>Let’s face it, scientific papers aren’t exactly page turners. They are written by scientists, for scientists, and often in a language that seems to only vaguely resemble English. </p>
<p>And perhaps one of the most daunting aspects of a scientific paper is the statistics (“stats”) section. </p>
<p>But what do stats really mean in the real world? Here’s an example from leukaemia research to help you break it down. </p>
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Read more:
<a href="https://theconversation.com/our-survey-found-questionable-research-practices-by-ecologists-and-biologists-heres-what-that-means-94421">Our survey found 'questionable research practices' by ecologists and biologists – here's what that means</a>
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<h2>Strength of results</h2>
<p>Stats are key to good research – they help researchers determine whether the results observed are strong enough to be due to an important scientific phenomenon. </p>
<p>As a research student I would always look for the magic number which indicates statistically significant differences in my experiments: most people agree this number to be 0.05 (you may see this in a paper written as p < 0.05). </p>
<p>When comparing two groups in a scientific study, statistical significance indicated by a p-value of less than 0.05 means that, in the case where there was no real difference between groups, there’s less than a 5% chance of the observed result arising. </p>
<p>But the focus on looking for <em>statistically</em> significant differences can blind us to the bigger picture. As I advanced through my scientific training I learnt to look for <em>biologically</em> significant differences. </p>
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Read more:
<a href="https://theconversation.com/the-curious-case-of-the-missing-workplace-teaspoons-103989">The curious case of the missing workplace teaspoons</a>
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<h2>Biological significance</h2>
<p>Biological significance addresses the question of whether the statistical difference actually means anything in terms of a real outcome, like a disease. Can the result explain how the disease is caused? Does it provide a new avenue to treat the disease? Basically, is it relevant?</p>
<p>A <a href="https://www.nature.com/articles/leu2016360">recent paper</a> published in the journal Leukaemia will help explain my point. The paper looked at why some people are able to stop their treatment for chronic myeloid leukaemia without the cancer coming back, while in others the cancer relapsed. </p>
<p>The key finding of this study was that patients who did not relapse had a higher proportion of natural killer cells compared to patients that did relapse. </p>
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<p><a href="https://www.immunology.org/public-information/bitesized-immunology/cells/natural-killer-cells">Natural killer cells</a> are a type of immune cell that controls viral infections and tumours. So, the more cells there are to kill the cancer, the less likely the cancer was to relapse – makes sense! </p>
<p>This finding has the potential to guide doctors in seeing which patients are likely to remain cancer-free after stopping treatment. This is definitely biologically significant.</p>
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Read more:
<a href="https://theconversation.com/my-cancer-is-in-remission-does-this-mean-im-cured-95429">My cancer is in remission – does this mean I'm cured?</a>
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<h2>Not so relevant</h2>
<p>Another result from the same paper (Figure 3a if you want to <a href="https://www.nature.com/articles/leu2016360">click through</a> to the data) shows a statistically significant difference in a sub-type of natural killer cells (called adaptive natural killer cells). But is this difference biologically relevant? </p>
<p>At this stage there is little evidence of a role for adaptive natural killer cells in the context of leukaemia. Also, the difference between the groups is relatively small, with a large variation within the groups (there are large <a href="https://www.biologyforlife.com/interpreting-error-bars.html">error bars</a> on the graph). </p>
<p>These factors make it more likely that the differences may be due to the mathematics involved in the statistical test rather than a biological effect. As with any new finding, time and further studies will be vital in working out whether this result actually means anything.</p>
<h2>Act like an expert</h2>
<p>So how do you pick if the statistical differences have biological value? Being a highly trained expert in the field certainly helps. </p>
<p>Another way to determine if the findings in a paper have biological relevance is to look for other papers that show similar results. If a result is “real” it should be found by other scientists who will build on it and publish more papers. </p>
<p>This means there will be lots of papers for you to read and apply your new-found passion for statistics.</p>
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<p><em>The open access research paper for this analysis is <a href="https://www.nature.com/articles/leu2016360">Increased proportion of mature NK cells is associated with successful imatinib discontinuation in chronic myeloid leukemia</a>.</em></p>
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<p><em>The definition of statistical significance has been edited since this article was first published.</em></p><img src="https://counter.theconversation.com/content/103329/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Yazad Irani 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>What do stats really mean in the real world? Here’s an example from leukaemia research to help you identify if a result really is important.Yazad Irani, Post-Doctoral Researcher, South Australian Health & Medical Research InstituteLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/1001252018-07-26T12:41:32Z2018-07-26T12:41:32ZCAR-T therapy works for some blood cancers, but can we make it work for brain tumours?<figure><img src="https://images.theconversation.com/files/228590/original/file-20180720-142426-qaio0n.jpg?ixlib=rb-1.1.0&rect=0%2C381%2C3244%2C2022&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">T-cells (red) attacking cancer cells (white).</span> <span class="attribution"><a class="source" href="https://www.shutterstock.com/download/confirm/770352868?src=MYlLj8JQfUKL9hrcO1Jwyw-1-8&size=huge_jpg">Meletios Verras/Shutterstock.com</a></span></figcaption></figure><p>A powerful new anti-cancer therapy, called CAR-T, has shown great promise in treating blood cancers. Last year, the Food and Drug Administration approved the treatment for acute lymphoblastic leukaemia, and the results have been very promising, with <a href="https://www.nejm.org/doi/10.1056/NEJMoa1709919">remission rates of up to 83%</a> for the very worst case patients, whose disease had returned following conventional treatments.</p>
<p>CAR-T (chimeric antigen receptor T-cell) therapy uses the body’s immune system to destroy cancer cells. It involves taking a patient’s T-cells (a type of immune cell) and genetically altering them so they attack cancer cells rather than invading organisms, such as bacteria. Large numbers of these CAR-T cells are grown in the lab before being infused into the patient.</p>
<p>With the recent success in mind, it is tempting to consider whether this approach would work in other cancers, particularly cancers that are notoriously difficult to treat, such as brain tumours. Unfortunately, this has not happened yet, and the reason is to do with the nature of cancer itself. </p>
<p>Cancer is not just one disease but many, each requiring a different treatment, and brain tumour treatment has a dismal history, with high-grade glioblastoma patients having an <a href="https://www.sciencedirect.com/science/article/pii/S2452336417300559">average survival rate of one year</a> after diagnosis.</p>
<h2>Why so difficult?</h2>
<p>So what is the problem with treating solid tumours and brain tumours in particular? The answer lies in the nature of the disease and the treatment. In leukaemia, there is a single, identifiable target: a molecule on the surface of the tumour cell called CD19. It’s a relatively straightforward task to engineer a CAR-T cell to attack that target and destroy the tumour. </p>
<p>In high-grade brain cancers (where cancer cells spread and grow more quickly), the tumour is heterogeneous, which means the target molecules on the tumour cells are constantly changing over the course of the disease. In different areas of the same tumour you will have different target molecules, so creating a CAR-T cell to attack the tumour becomes an impossible task. </p>
<p>Also, high-grade brain tumours build a network of blood vessels to support and feed the tumour as it grows and spreads to other areas of the brain, in a process known as “angiogenesis”. Understandably, this makes some brain tumours very difficult to treat and remove.</p>
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<img alt="" src="https://images.theconversation.com/files/228589/original/file-20180720-142438-1s2gioo.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/228589/original/file-20180720-142438-1s2gioo.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=450&fit=crop&dpr=1 600w, https://images.theconversation.com/files/228589/original/file-20180720-142438-1s2gioo.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=450&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/228589/original/file-20180720-142438-1s2gioo.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=450&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/228589/original/file-20180720-142438-1s2gioo.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=566&fit=crop&dpr=1 754w, https://images.theconversation.com/files/228589/original/file-20180720-142438-1s2gioo.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=566&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/228589/original/file-20180720-142438-1s2gioo.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">MRI showing brain cancer.</span>
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<p>Another feature of brain tumours involves another type of immune cell called the regulatory T-cell. These are the cells that stop our immune system from attacking our own bodies. (When they go wrong they cause autoimmune diseases such as rheumatoid arthritis.) Brain tumours can hijack these cells to create an immunosuppressive micro-environment for the tumour to live in. Even if immune cells could get to the site of the tumour, they would be “switched off” by the actions of these regulatory T-cells, and the tumour would be safely protected from the attack.</p>
<h2>Dismantling safe spaces</h2>
<p>The outlook for using immunotherapy to treat brain cancer looks poor with several barriers to be overcome, but let’s look again at CAR-T therapy. </p>
<p>We can create a specific immune cell to attack a specific target and fourth-generation CAR-T cells can be produced to attack two or more specific targets – but this isn’t enough. So instead of focusing on the moving target of the tumour cell, perhaps we should look at other targets. </p>
<p>The processes of angiogenesis (the development of new blood vessels) and immuno-suppression are driven by cells that have their own unique molecular characteristics, so let’s target these areas. Let’s take away the “safe space” the tumour has created for itself by destroying the micro-environment of the tumour, allowing our immune cells to do their job of destroying it.</p>
<p>There is a major problem that would need to be overcome for this to work, which is systemic toxicity – the result of the engineered CAR-T cells attacking the wrong healthy target cells. The last thing we need is for our CAR-T cells to destroy new blood vessels or all regulatory T cells.</p>
<p>What we need are tumour-specific targets for the newly created blood vessels and the immune-suppressing T-cells. Finding these will be the focus of my future research.</p><img src="https://counter.theconversation.com/content/100125/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Peter Abel 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>CAR-T is a powerful new immunotherapy for treating leukaemia. We need to build on it to attack other, harder-to-treat cancers.Peter Abel, Senior lecturer in haematology, immunolgy, genetics and evolution, University of Central LancashireLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/900672018-01-15T16:06:50Z2018-01-15T16:06:50ZRevealed: adult leukaemia can be caused by gene implicated in breast cancer and obesity<figure><img src="https://images.theconversation.com/files/201962/original/file-20180115-101505-1tj72tw.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">AML under the microscope. </span> <span class="attribution"><a class="source" href="https://www.shutterstock.com/image-photo/blood-smear-under-microscopy-showing-on-550330186?src=1RRsQj8cvHJjbcMN_p5BAw-1-37">Medtech THAI STUDIO LAB 249</a></span></figcaption></figure><p>When people think of leukaemia, they usually think of blood cancers that affect children. These mostly come under the category of acute lymphoblastic leukaemia – or ALL – and are different to the group of blood cancers which predominantly affect adults over the age of 60, known as acute myeloid leukaemia (AML). </p>
<p>AML accounts for about 90% of all leukaemias in adults, though it affects some children too. With <a href="http://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/leukaemia-aml/incidence#heading-One">some 3,000</a> new cases each year in the UK alone, it is tougher to treat than ALL. </p>
<p>Where advances in ALL treatment have raised survival rates to <a href="https://www.stjude.org/disease/acute-lymphoblastic-leukemia-all.html">around 90%</a> over the past several decades, the rates for surviving the less well researched AML are <a href="https://www.healthline.com/health/acute-myeloid-leukemia-survival-rates-outlook">more like</a> 65%. Older adults respond least well to treatment, with only 5% of over-65s surviving more than five years. </p>
<p>I am therefore pleased to report a promising discovery. Work in which I have been involved has shown that a particular gene can play a critical role in the development of the disease. This could be the precursor to a breakthrough that could be life-saving for patients. </p>
<h2>Cells and treatments</h2>
<p>Your bone marrow contains stem cells which divide and differentiate into red blood cells and the main groups of white blood cells – myeloid cells, neutrophils and lymphocytes. Normally this happens in a very controlled manner, ensuring you have all the red blood cells needed to carry oxygen around your body, and all the white blood cells needed to fight off infections. </p>
<p>In AML too many immature myeloid cells are produced too quickly by the bone marrow. They are mutant cells which don’t mature, meaning they fail to defend against infection. </p>
<p>For this reason, early signs of AML include flu-like symptoms, aches and pains in the joints, and rapid weight loss. As the abnormal cells build up inside the bone marrow or the blood they grow and divide aggressively. Left untreated, AML patients can have only weeks to live. </p>
<figure class="align-right zoomable">
<a href="https://images.theconversation.com/files/201963/original/file-20180115-101505-e9kfw7.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/201963/original/file-20180115-101505-e9kfw7.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=237&fit=clip" srcset="https://images.theconversation.com/files/201963/original/file-20180115-101505-e9kfw7.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=913&fit=crop&dpr=1 600w, https://images.theconversation.com/files/201963/original/file-20180115-101505-e9kfw7.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=913&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/201963/original/file-20180115-101505-e9kfw7.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=913&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/201963/original/file-20180115-101505-e9kfw7.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=1148&fit=crop&dpr=1 754w, https://images.theconversation.com/files/201963/original/file-20180115-101505-e9kfw7.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=1148&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/201963/original/file-20180115-101505-e9kfw7.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=1148&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
<figcaption>
<span class="caption">Bone marrow transplant.</span>
<span class="attribution"><a class="source" href="https://www.shutterstock.com/image-photo/bone-marrow-transplant-operation-786989008?src=V3Gd5UZImFRTdR5V6xaclg-1-7">El-Roi</a></span>
</figcaption>
</figure>
<p>Patients are normally treated by two stages of chemotherapy over a few months: an induction phase which reduces the number of cancer cells to undetectable levels, then a consolidation phase to kill any cancerous cells hiding in the body. Patients often also receive a bone marrow transplant, effectively giving them a new immune system. </p>
<p>Treating AML is complicated by patients generally being older, since they tolerate the intensive chemotherapy less well. In many cases, they receive some treatment and end up only living a few months. Better understanding the mutations to develop more targeted and less harsh treatments looks like the key to improving survival. </p>
<h2>Step forward, PTPN1</h2>
<p>A number of mutations are associated with AML, and often occur in combinations. It’s these mixtures of mutations that are thought to cause the <a href="https://www.cancer.org/cancer/acute-myeloid-leukemia/detection-diagnosis-staging/how-classified.html">complex subtypes</a> of cancers within the AML group. One common mutation is called <a href="http://www.cytocell.com/probes/23-del20q-deletion">Del20q</a>. It involves the deletion of part of <a href="https://ghr.nlm.nih.gov/chromosome/20">chromosome 20</a>, one of the 23 pairs of chromosomes most humans have in all their cells. </p>
<p>It has <a href="http://www.bloodjournal.org/content/bloodjournal/82/11/3424.full.pdf?sso-checked=true">long been suspected</a> that genes on this part of the chromosome may function, either individually or together, to suppress cancer. Until recently, however, researchers have found it hard to say which genes are responsible. </p>
<p>One candidate is known as PTPN1, or protein tyrosine phosphatase, non-receptor type 1. First discovered in the late 1980s and linked to metabolic function, it is more famously known for its roles in <a href="https://www.ncbi.nlm.nih.gov/pubmed/27465552">breast cancer</a> and <a href="https://www.ncbi.nlm.nih.gov/pubmed/25120222">type 2 diabetes</a>. Its location on chromosome 20 has long made specialists suspect it could also be involved in AML. </p>
<p>It was <a href="https://www.nature.com/articles/leu201731">shown recently</a> that when you switch off the equivalent gene in mice, it leads to what are known as <a href="https://www.cancersupportcommunity.org/myeloproliferative-neoplasms">myeloproliferative neoplasm</a>, which is the wider family of blood cancers of which AML is a member. In <a href="http://cancerres.aacrjournals.org/content/early/2017/11/09/0008-5472.CAN-17-0946">our new study</a>, we have taken this a step forward: we have shown that if you delete this gene in older mice, it specifically gives rise to AML – and in a similar way to how the disease develops in older humans. </p>
<p>The previous study showed that PTPN1 is deleted from chromosome 20 in the cells of patients in around 17% of AML cases, which raises questions about the remaining majority of cases. We were able to show that deleting the mouse equivalent of PTPN1 activates a molecule called STAT3, which is important to regulating cell growth and division. </p>
<p>If a patient has too much STAT3, it leads to the generation of too many immature myeloid cells – that hallmark of AML I mentioned earlier. This is potentially a very useful finding for further studies into the genetics behind the disease: in the two other most common mutations linked to AML, which relate to a protein called JAK2 and a receptor called FLT-3, STAT3 is also over-activated. In all, STAT3 is relevant to maybe three quarters of all AML cases. Uncovering exactly how they relate looks critical to developing an eventual cure. </p>
<h2>The future</h2>
<p>In short, we’re closing in on understanding the links between PTPN1, STAT3 and AML. A few years from now, as the cost of genome sequencing falls, it will become a question of identifying which combination of mutations has affected a patient and prescribing a treatment accordingly. </p>
<p>This treatment will probably be more bespoke chemotherapy for patients that can tolerate it, and perhaps gene editing using tools such as <a href="https://www.broadinstitute.org/what-broad/areas-focus/project-spotlight/questions-and-answers-about-crispr">CRISPR</a> for those that cannot. Doctors would edit the correct versions of genes like PTPN1 back into the patient’s bone marrow, potentially restoring normal function and negating the often difficult search for a compatible bone marrow donor. </p>
<figure class="align-center zoomable">
<a href="https://images.theconversation.com/files/201964/original/file-20180115-101498-q5v593.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/201964/original/file-20180115-101498-q5v593.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/201964/original/file-20180115-101498-q5v593.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=400&fit=crop&dpr=1 600w, https://images.theconversation.com/files/201964/original/file-20180115-101498-q5v593.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=400&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/201964/original/file-20180115-101498-q5v593.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=400&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/201964/original/file-20180115-101498-q5v593.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=503&fit=crop&dpr=1 754w, https://images.theconversation.com/files/201964/original/file-20180115-101498-q5v593.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=503&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/201964/original/file-20180115-101498-q5v593.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>
<figcaption>
<span class="caption">New edition.</span>
<span class="attribution"><a class="source" href="https://www.shutterstock.com/image-photo/bone-marrow-transplant-operation-786989008?src=V3Gd5UZImFRTdR5V6xaclg-1-7">vchal</a></span>
</figcaption>
</figure>
<p>There is much research still to be done. We need to understand what PTPN1 is doing in healthy myeloid cells to grasp which processes are disturbed when it becomes deleted. The other big question is whether instead of getting deleted, PTPN1 sometimes more subtly mutates and how this relates to AML. Besides this, there are many other genes on the Del20q deletion that we need to better understand, too. </p>
<p>In the meantime, showing that removing PTPN1 leads to AML is an important piece of the puzzle. It brings the day closer when survival rates for AML make the same climb that we have seen in other kinds of leukaemia, and hopefully even beyond.</p><img src="https://counter.theconversation.com/content/90067/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Samantha Le Sommer 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>Improvements in survival rates for acute myeloid leukaemia have failed to keep pace with other leukaemias. That may be about to change.Samantha Le Sommer, Postdoctoral Researcher, University of AberdeenLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/821002017-08-08T12:20:46Z2017-08-08T12:20:46ZA revolutionary leukaemia treatment could soon be approved – here’s what it means for patients<figure><img src="https://images.theconversation.com/files/181162/original/file-20170807-16718-1jb7pf7.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">DNA: the new battleground.</span> <span class="attribution"><a class="source" href="https://www.shutterstock.com/download/success?src=SjcP5lDOkJc4evPsgIArcQ-1-99">Shutterstock</a></span></figcaption></figure><p>A revolutionary gene <a href="http://www.nejm.org/doi/full/10.1056/NEJMoa1407222#t=article">therapy treatment for leukaemia</a>
has been recommended by an advisory panel for approval by the Food and Drug Administration (FDA), the regulatory body in the US.</p>
<p>If the FDA approves the treatment – it is expected to take a decision in September – CTL019 will be the first gene therapy allowed for clinical use in the US and the fourth globally, following <a href="http://www.uniqure.com/gene-therapy/glybera.php">Glybera</a>
and <a href="https://hcp.gsk.co.uk/products/strimvelis.html">Strimvelis</a>, two gene therapy products recently approved in Europe for two genetic diseases, and <a href="https://www.ncbi.nlm.nih.gov/pubmed/16149900">Gendicine</a> (for cancer) in China in 2004. </p>
<p>CTL019 is a custom-made, personalised drug developed by the University of Pennsylvania and health company Novartis. The treatment consists of a single shot of immune cells reprogrammed to kill the patient’s specific leukemic cells. </p>
<p>Clinical trials have shown remarkable results. Originally <a href="http://www.nejm.org/doi/full/10.1056/NEJMoa1407222#t=article">tested in 30 patients</a> affected by resistant acute <a href="http://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/types">B-lymphoblastic leukaemia (ALL)</a> – an aggressive blood cancer – CTL019 induced complete remission (meaning that the leukaemia vanished) in 90% of patients. The trial was later extended to <a href="https://ash.confex.com/ash/2016/webprogram/Paper90831.html">50 patients</a> and resulted in a similar remission rate, which is extremely high for ALL. </p>
<p>ALL <a href="http://onlinelibrary.wiley.com/doi/10.1111/bjh.13852/abstract">accounts for 25%</a> of all cancer in children. The standard treatment is based on chemotherapy followed by bone marrow transplantation. If the standard treatment fails, a bone marrow donor is unavailable or the patient relapses, there are no other therapeutic options and the chances of survival are slim. It is <a href="http://onlinelibrary.wiley.com/doi/10.1111/bjh.13852/abstract">estimated that 15%</a> of cases do not respond to standard treatment. </p>
<p>CTL019 represents a major opportunity for these children. Indeed, families of children affected by ALL, whose lives were <a href="http://www.nejm.org/doi/full/10.1056/NEJMoa1407222#t=article">saved by CTL019</a>, strongly <a href="https://ash.confex.com/ash/2016/webprogram/Paper90831.html">advocated</a> for its approval. </p>
<h2>How it works</h2>
<p>The treatment is the result of over 20 years of research in the fields of molecular biology, virology and haematology. </p>
<p>The administration of CTL019 is a lengthy procedure. Immune cells called <a href="https://www.britannica.com/science/T-cell">T cells</a> are taken from the patient using a procedure similar to blood collection. The cells are then grown and genetically modified in a laboratory for expression of a molecule receptor called CAR (chimeric antigen receptor) which is able to recognise CD19, a molecule widely expressed on B-leukemic cells. The genetically-modified immune cells are then administered back to the patient in a procedure which resembles a transfusion. </p>
<figure class="align-center ">
<img alt="" src="https://images.theconversation.com/files/181163/original/file-20170807-16741-1phmmdv.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/181163/original/file-20170807-16741-1phmmdv.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=400&fit=crop&dpr=1 600w, https://images.theconversation.com/files/181163/original/file-20170807-16741-1phmmdv.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=400&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/181163/original/file-20170807-16741-1phmmdv.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=400&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/181163/original/file-20170807-16741-1phmmdv.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=503&fit=crop&dpr=1 754w, https://images.theconversation.com/files/181163/original/file-20170807-16741-1phmmdv.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=503&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/181163/original/file-20170807-16741-1phmmdv.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">A new way to fight leukaemia.</span>
<span class="attribution"><a class="source" href="https://www.shutterstock.com/download/success?src=YrLzQ3vUklbiXnwckl4vmA-1-26">Shutterstock</a></span>
</figcaption>
</figure>
<p>It is unclear how long the CTL019 reprogrammed cells will persist in the patients’ circulation and whether this should be a reason to worry or celebrate. It is not fully understood whether they will remain for long periods of time and go on to harm the patient, for example, or persist and offer protection from any further leukemic growth.</p>
<p>In the lab, CTL019 has been added to T cells by inserting specific genes inside the T cells using viruses as vectors for the genetic transfer. This is also the system used in the Strimvelis and Glybera treatments. </p>
<p>This approach exploits the natural ability of certain viruses to infect cells and introduce genes into them. The vectors used are not virulent and therefore they are not able to cause a disease, but they can add genes to the human genome. </p>
<p>The first trials using this type of approach didn’t fully take into consideration the potential harm of inserting a gene into a random sequence of human DNA. The results had a tragic setback: in 2002, a gene therapy <a href="http://www.nature.com/nature/journal/v420/n6912/full/420116a.html?foxtrotcallback=true">patient developed a leukaemia</a>. This had a profound impact on public opinion.</p>
<p>Since then, enormous efforts have been made to improve the methodology and vector design in order to reduce the risks. Nevertheless, the long-term effects of such new treatments are unknown and patients will need to be under close monitoring for 15 years. </p>
<p>As scientists, we all hope and work hard to find a definitive cure for all cancers. We don’t know yet if the patients that undertook the CTL019 trials can be considered cured or whether they will eventually relapse. But this drug is an opportunity to prolong the survival of patients that currently have no other option. In the meantime, we will celebrate every success in the battle to prolong the survival of cancer patients. </p>
<p>An analogous protocol called <a href="http://labiotech.eu/ucart19-universal-car-t-given-to-another-baby-gosh-cellectis-leukemia/">UCART19</a>, is also under development at the Great Ormond Street Hospital in London. In 2015, a one-year-old baby affected by refractory ALL was the first patient <a href="http://www.gosh.nhs.uk/news/latest-press-releases/2015-press-release-archive/world-first-use-gene-edited-immune-cells-treat-incurable-leukaemia">ever treated with UCART19</a>. The brilliant results led to the development of a <a href="http://www.gosh.nhs.uk/research-and-innovation/nihr-great-ormond-street-brc/brc-news/clinical-trial-investigate-treatment-acute-lymphoblastic-leukaemia-open-gosh">phase I clinical trial</a>. </p>
<p>No treatment can be considered 100% safe. For every patient, there are risks and benefits to take into consideration. Nevertheless, CTL019 has produced some excellent results and the side effects have been managed well. At the moment, this treatment is a very promising option for patients with relapsed ALL. Let’s hope it saves lives.</p><img src="https://counter.theconversation.com/content/82100/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Maria Teresa Esposito 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 gene therapy CTL019 induced complete remission in 90% of patients.Maria Teresa Esposito, Lecturer in Biomedical Science (Cancer biology), University of East LondonLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/803272017-07-26T20:15:02Z2017-07-26T20:15:02ZBlood tests and diagnosing illness: what can blood tell us about what’s happening in our body?<figure><img src="https://images.theconversation.com/files/176715/original/file-20170704-12293-nwqqm7.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Blood permeates every tissue in the body, meaning just a few spoonfuls can tell us a lot about our health. </span> <span class="attribution"><span class="source">from www.shutterstock.com.au</span></span></figcaption></figure><p><em>This week we’re running a series in collaboration with the Australian Red Cross Blood Service looking at blood: what it actually does, why we need it, and what happens when something goes wrong. Read other articles in the series <a href="https://theconversation.com/au/topics/blood-series-39533">here</a>.</em></p>
<hr>
<p>Doctors have continually sought better ways of determining what is wrong with a patient. When you visit a GP’s office or emergency department with an unknown illness, a doctor will commonly draw some blood to gain a better idea of what’s going on inside your body. Blood is perhaps the most important window through which we can peer into a person’s health or illness.</p>
<p>About 7% of our body weight is our blood, and our heart spits out about five litres of blood every minute. Oxygenated blood leaves the left side of the heart via the aorta and the arteries - which permeate every tissue in the body - and returns to the right side of the heart via the veins. From the right side of the heart, blood is pumped into the lungs where it is oxygenated, returning to the left side of the heart.</p>
<p>In about two tablespoons of blood there’s a lot we can tell about our health.</p>
<h2>What blood can tell us</h2>
<p>When someone presents at an emergency department, the initial panel of tests will include a full blood count. This details the red blood cell count, white blood cell count and platelets; electrolytes (the substance in our blood that carries an electric charge that is vital for life) to measure kidney function; liver function tests and “C-reactive protein” which can tell us if there is inflammation somewhere in the body.</p>
<figure class="align-center zoomable">
<a href="https://images.theconversation.com/files/177464/original/file-20170710-5553-yu4kic.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/177464/original/file-20170710-5553-yu4kic.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/177464/original/file-20170710-5553-yu4kic.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=400&fit=crop&dpr=1 600w, https://images.theconversation.com/files/177464/original/file-20170710-5553-yu4kic.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=400&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/177464/original/file-20170710-5553-yu4kic.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=400&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/177464/original/file-20170710-5553-yu4kic.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=503&fit=crop&dpr=1 754w, https://images.theconversation.com/files/177464/original/file-20170710-5553-yu4kic.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=503&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/177464/original/file-20170710-5553-yu4kic.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>
<figcaption>
<span class="caption">Our blood can tell us how many of our organs are functioning.</span>
<span class="attribution"><span class="source">from www.shutterstock.com</span></span>
</figcaption>
</figure>
<p>From these tests we can determine things like the presence of anaemia (low red blood cells from various causes), infection, kidney failure and liver disease. Often the results of these tests will trigger further testing. For example, the presence of anaemia will usually lead to testing for iron deficiency, possibly vitamin B12 and folate, screening for haemolysis (destruction of red blood cells) and a measure of how well the bone marrow, which makes red blood cells, is responding. </p>
<p>If infection is suspected, blood will be drawn and transferred into a bottle that enables bacteria to grow. Bacteria in the blood is called septicaemia. The identification of the bacteria responsible significantly helps in the management, meaning the right antibiotics can be delivered to the patient.</p>
<hr>
<p><a href="https://theconversation.com/from-animal-experiments-to-saving-lives-a-history-of-blood-transfusions-80391"><em>Infographic - From animal experiments to saving lives: a history of blood transfusions</em></a></p>
<hr>
<p>Bruising or excessive bleeding will prompt assessment of platelets and clotting. Platelets are the first responders to injury, and if they are low or not functioning properly, they will allow bleeding to proceed unchecked. To tell if blood is clotting normally we need an additional teaspoon of blood. These clotting factors are synthesised in the liver, so they can also give us a warning about liver disease.</p>
<p>As a kidney specialist, my personal favourite are the electrolytes. Together with a urine test, blood electrolytes can measure someone’s kidney disease from stage one through to five. As kidney function declines, potassium levels increase in the blood and can reach dangerous levels. A high potassium count can cause a potentially fatal heart arrhythmia.</p>
<p>Liver function tests provide information on what the liver is producing and excreting - abnormalities of liver function could mean gall stones or hepatitis. Viral causes of hepatitis, such as Hepatitis B and C, can quickly be checked in the blood. We can also find out how recently the infection was acquired and whether chronic infection persists.</p>
<p>Cardiac enzymes in the blood tell us if a patient has had a heart attack. The enzymes are proteins released from damaged heart muscle, so the higher the level, the greater the damage to the heart.</p>
<figure class="align-center zoomable">
<a href="https://images.theconversation.com/files/179116/original/file-20170721-30878-unhpag.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/179116/original/file-20170721-30878-unhpag.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/179116/original/file-20170721-30878-unhpag.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=400&fit=crop&dpr=1 600w, https://images.theconversation.com/files/179116/original/file-20170721-30878-unhpag.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=400&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/179116/original/file-20170721-30878-unhpag.jpg?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=400&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/179116/original/file-20170721-30878-unhpag.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=503&fit=crop&dpr=1 754w, https://images.theconversation.com/files/179116/original/file-20170721-30878-unhpag.jpg?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=503&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/179116/original/file-20170721-30878-unhpag.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>
<figcaption>
<span class="caption">Blood is an incredible window into the workings of the human body.</span>
<span class="attribution"><span class="source">Blood on Silk / Buy Sell by Fiona Davies (AUS) Courtesy of the artist as part of Science Gallery Melbourne’s BLOOD exhibition</span></span>
</figcaption>
</figure>
<h2>What can’t blood tell us?</h2>
<p>The repertoire of blood tests available to the treating doctor is vast. Other blood tests include screening for autoimmune disease, monitoring the response of cancers to treatment with tumour markers, assessing reproductive function, screening for genetic disorders during pregnancy and diagnosing pregnancy itself.</p>
<p>Despite this, sometimes a diagnosis remains elusive – frustrating for the patient and doctor alike. Neurological disease such as stroke, motor neurone disease, Alzheimer’s and multiple sclerosis aren’t diagnosable from blood tests. Similarly, the diagnoses of depression, schizophrenia, ADHD and autism lack a specific blood diagnostic marker.</p>
<p>The huge array of blood tests available to the clinician aid in a rapid diagnosis in many instances. But the choice and the interpretation of the test needs to be considered in light of the patient and their presenting symptoms. As the old adage in medicine says: treat the person and not the numbers.</p>
<hr>
<p><em><strong>Read other articles in the series:</strong></em></p>
<p><em><a href="https://theconversation.com/essays-on-blood-why-do-we-actually-have-it-75064">Essays on blood: why do we actually have it?</a></em></p>
<p><em><a href="http://theconversation.com/from-animal-experiments-to-saving-lives-a-history-of-blood-transfusions-80391">From animal experiments to saving lives: a history of blood transfusions</a></em></p>
<p><em><a href="http://theconversation.com/explainer-whats-actually-in-our-blood-75066">Explainer: what’s actually in our blood?</a></em></p>
<p><a href="http://theconversation.com/blood-groups-beyond-a-b-and-o-what-are-they-and-do-they-matter-75063"><em>Blood groups beyond A, B and O: what are they and do they matter?</em></a></p>
<p><em><a href="http://theconversation.com/what-can-go-wrong-in-the-blood-a-brief-overview-of-bleeding-clotting-and-cancer-76400">What can go wrong in the blood? A brief overview of bleeding, clotting and cancer</a></em></p><img src="https://counter.theconversation.com/content/80327/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Karen Dwyer 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>Blood is perhaps the most important window through which we can peer into a person’s health or illness.Karen Dwyer, Deputy Head, School of Medicine, Deakin UniversityLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/806082017-07-07T05:40:37Z2017-07-07T05:40:37ZPrivate clinics’ peddling of unproven stem cell treatments is unsafe and unethical<figure><img src="https://images.theconversation.com/files/177253/original/file-20170707-25361-a3a7in.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Stem cells have saved thousands of lives thanks to their applications in cancer treatments. Many other uses peddled by private clinics are without evidence. </span> <span class="attribution"><span class="source">from www.shutterstock.com</span></span></figcaption></figure><p>Stem cell science is an area of medical research that continues to offer great promise. But as this week’s paper in <a href="http://stm.sciencemag.org/content/9/397/eaag0426">Science Translational Medicine</a> highlights, a growing number of clinics around the globe, including in Australia, are exploiting regulatory gaps to sell so-called stem cell treatments without evidence that what they offer is effective – or even safe.</p>
<p>Such unregulated direct-to-consumer advertising – typically of cells obtained using liposuction-like methods – not only places the health of individuals at risk, but could also undermine the legitimate development of stem cell-based therapies. </p>
<p>Many academic societies and professional medical organisations have <a href="http://www.nature.com/news/policy-global-standards-for-stem-cell-research-1.19908">raised concerns</a> about these futile and often expensive cell therapies. Despite this, national regulators have typically been slow or ineffective in curtailing them. </p>
<p>As well as tighter regulations here, international regulators such as the World Health Organisation and the International Council on Harmonisation need to move on ensuring patients desperate for cures aren’t sold treatments with limited efficacy and unknown safety.</p>
<h2>So what’s on offer?</h2>
<p><a href="http://www.cell.com/cell-stem-cell/abstract/S1934-5909(16)30210-7">Hundreds of stem cell clinics</a> post online claims that they have been able to treat patients suffering from a wide range of conditions. These include osteoarthritis, pain, spinal cord injury, multiple sclerosis, diabetes and infertility. The websites are high on rhetoric of science – often using various accreditation, awards and other tokens to imply legitimacy – but low on proof that they work. </p>
<p>Rather than producing independently verified results, these clinics rely on patient testimonials or unsubstantiated claims of “improvement”. In so doing these shonky clinics understate the risks to patient health associated with these unproven stem cell-based interventions. </p>
<figure class="align-center zoomable">
<a href="https://images.theconversation.com/files/177247/original/file-20170706-17238-a66fxh.png?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/177247/original/file-20170706-17238-a66fxh.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/177247/original/file-20170706-17238-a66fxh.png?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=309&fit=crop&dpr=1 600w, https://images.theconversation.com/files/177247/original/file-20170706-17238-a66fxh.png?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=309&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/177247/original/file-20170706-17238-a66fxh.png?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=309&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/177247/original/file-20170706-17238-a66fxh.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=389&fit=crop&dpr=1 754w, https://images.theconversation.com/files/177247/original/file-20170706-17238-a66fxh.png?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=389&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/177247/original/file-20170706-17238-a66fxh.png?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=389&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 cell clinics often rely on anecdotes and patient ‘testimonials’.</span>
<span class="attribution"><a class="source" href="http://www.startstemcells.com/?gclid=CKDX2Pfq9dQCFVYFKgodGHIP9g">Screenshot, Swiss Medica website</a></span>
</figcaption>
</figure>
<p>Properly administered informed consent is often overlooked or ignored, so patients can be misled about the likelihood of success. In addition to heavy financial burdens imposed on patients and their families, there is often an “opportunity cost” because the time wasted in receiving futile stem cells diverts patients away from proven medicines.</p>
<p>The many recent reports of adverse outcomes demonstrate the risks of receiving unproven cell therapies are not trivial. In the USA <a href="https://www.washingtonpost.com/news/to-your-health/wp/2017/03/15/three-women-blinded-by-unapproved-stem-cell-treatment-at-south-florida-clinic/?utm_term=.92e3f1212d38">three women were blinded</a> following experimental “stem cell” treatment for macular degeneration (a degenerative eye disease that can cause blindness). One <a href="https://www.nytimes.com/2016/06/23/health/a-cautionary-tale-of-stem-cell-tourism.html">man was rendered a quadriplegic</a> following a stem cell intervention for stroke. And a woman whose family sought treatment for her dementia <a href="http://www.abc.net.au/radionational/programs/backgroundbriefing/hallmarks-of-quack-medicine-in-fatal-stem-cell-treatment/7630288">died in Australia</a>. </p>
<figure class="align-center zoomable">
<a href="https://images.theconversation.com/files/177248/original/file-20170706-18915-31hviz.png?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/177248/original/file-20170706-18915-31hviz.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/177248/original/file-20170706-18915-31hviz.png?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=511&fit=crop&dpr=1 600w, https://images.theconversation.com/files/177248/original/file-20170706-18915-31hviz.png?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=511&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/177248/original/file-20170706-18915-31hviz.png?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=511&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/177248/original/file-20170706-18915-31hviz.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=642&fit=crop&dpr=1 754w, https://images.theconversation.com/files/177248/original/file-20170706-18915-31hviz.png?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=642&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/177248/original/file-20170706-18915-31hviz.png?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=642&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
<figcaption>
<span class="caption">Many clinics make claims stem cell treatments are effective for a vast range of conditions, most of which there is limited evidence for.</span>
<span class="attribution"><a class="source" href="http://www.stemcelltherapyplus.com/">Screenshot, Stem Cell Therapy Plus website</a></span>
</figcaption>
</figure>
<p>Other notorious cases involving the deaths of patients include the German government shutting down the <a href="http://www.eurostemcell.org/stem-cell-tourism-selling-hope-through-unproven-stem-cell-treatments-lessons-x-cell-center">X-Cell Centre</a> and the <a href="http://www.nature.com/news/disgraced-stem-cell-entrepreneur-under-fresh-investigation-1.20985">Italian government closing the Stamina Foundation</a> it had previously supported.</p>
<h2>What’s approved?</h2>
<p>At present, the only recognised stem cell treatments are those utilising blood stem cells isolated from bone marrow, peripheral blood (the cellular components of blood such as red and white blood cells and platelets) or umbilical cord blood. </p>
<p>Hundreds of thousand of <a href="http://www.cancerresearchuk.org/about-cancer/cancer-in-general/treatment/bone-marrow-stem-cell-transplants/stem-cell-transplants">lives have been saved</a> over the last half-century in patients with cancers such as leukaemia, lymphoma and multiple myeloma, as well as rare inherited immune and metabolic disorders. </p>
<p>A few types of cancer and autoimmune diseases may also benefit from blood stem cells in the context of chemotherapy. Different stem cells are also successfully used for <a href="http://www.nature.com/news/behind-the-scenes-of-the-world-s-first-commercial-stem-cell-therapy-1.17022">corneal</a> and <a href="http://www.eurostemcell.org/skin-stem-cells-where-do-they-live-and-what-can-they-do">skin grafting</a>. </p>
<p>All other applications remain in the preclinical research phase or are just starting to be evaluated in <a href="https://www.australianclinicaltrials.gov.au/what-clinical-trial">clinical trials</a>. </p>
<hr>
<p><em>Further reading: <a href="https://theconversation.com/yes-theres-hope-but-treating-spinal-injuries-with-stem-cells-is-not-a-reality-yet-72493">Yes there’s hope, but treating spinal injuries with stem cells is not a reality yet</a></em></p>
<hr>
<p>Often dismissed by for-profit clinics as “red tape” hampering progress, the rigour of clinical trials allows for the collection of impartial evidence. Such information is usually required before a new drug or medical device is released into the marketplace. Unfortunately, in the case of for-profit stem cell clinics, their marketing has gazumped the scientific evidence. </p>
<figure class="align-center zoomable">
<a href="https://images.theconversation.com/files/177252/original/file-20170706-14401-ocg5k5.png?ixlib=rb-1.1.0&q=45&auto=format&w=1000&fit=clip"><img alt="" src="https://images.theconversation.com/files/177252/original/file-20170706-14401-ocg5k5.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/177252/original/file-20170706-14401-ocg5k5.png?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=349&fit=crop&dpr=1 600w, https://images.theconversation.com/files/177252/original/file-20170706-14401-ocg5k5.png?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=349&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/177252/original/file-20170706-14401-ocg5k5.png?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=349&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/177252/original/file-20170706-14401-ocg5k5.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=438&fit=crop&dpr=1 754w, https://images.theconversation.com/files/177252/original/file-20170706-14401-ocg5k5.png?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=438&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/177252/original/file-20170706-14401-ocg5k5.png?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=438&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px"></a>
<figcaption>
<span class="caption">Marketing regulation is needed so private clinics can’t make claims without evidence.</span>
<span class="attribution"><a class="source" href="http://www.asctreatment.com.au">Screenshot, ASC Treatment website</a></span>
</figcaption>
</figure>
<h2>So what can be done?</h2>
<p>Action is required on many fronts. Regulators at both an international and national level need to tackle regulatory loopholes and challenge unfounded marketing claims of businesses selling unproven stem cell interventions. </p>
<p>Researchers <a href="https://theconversation.com/why-the-media-need-to-tread-carefully-when-reporting-research-findings-72074">need to more clearly communicate their findings</a> and the necessary next steps to responsibly take their science from the laboratory to the clinic. And they should acknowledge that this will take time. </p>
<p>Patients and their loved ones must be encouraged to seek advice from a trained reputable health care professional, someone who knows their medical history. They should think twice if someone is offering a treatment outside standards of practice. </p>
<p>The stakes are too high not to have these difficult conversations. If a stem cell treatment sounds too good to be true, it probably is.</p>
<hr>
<p><em>For more information on recognised stem cell treatments visit the <a href="http://www.stemcellfoundation.net.au/docs/patient-handbook/australian-stem-cell-handbook.pdf?sfvrsn=4">National Stem Cell Foundation of Australia and Stem Cells Australia</a>, <a href="https://www.choice.com.au/health-and-body/hospitals-and-medical-procedures/medical-treatments/articles/stem-cell-therapy#what%20conditions">Choice Australia</a>, <a href="http://www.eurostemcell.org/considering-stem-cell-treatment-offer">EuroStemCell</a>, <a href="http://www.closerlookatstemcells.org/">International Society for Stem Cell Research</a>, and <a href="http://www.celltherapysociety.org/?page=PTF2015">International Society for Cellular Therapy</a>.</em></p><img src="https://counter.theconversation.com/content/80608/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Megan Munsie receives funding from the Australian Research Council. She is affiliated with the International Society for Stem Cell Research, Australasian Society for Stem Cell Research and the International Society for Cellular Therapy. </span></em></p><p class="fine-print"><em><span>John Rasko receives funding from the Brocher Foundation and Cure The Future.</span></em></p>Stem cell science continues to offer great promise. But a growing number of clinics are selling treatments without evidence that what they offer is effective – or even safe.Megan Munsie, Deputy Director - Centre for Stem Cell Systems and Head of Education, Ethics, Law & Community Awareness Unit, Stem Cells Australia, The University of MelbourneJohn Rasko, Clinical Haematologist and President-Elect, International Society for Cellular Therapy., University of SydneyLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/770902017-05-04T01:46:00Z2017-05-04T01:46:00ZNew drugs on the PBS: what they do and why we need them<figure><img src="https://images.theconversation.com/files/167678/original/file-20170503-21637-4oi5od.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Some of the notable additions to the PBS include drugs to treat eye and HIV infections, cystic fibrosis, multiple sclerosis, and cancer.</span> <span class="attribution"><span class="source">from shutterstock.com</span></span></figcaption></figure><p>This week, the government <a href="http://www.pbs.gov.au/browse/changes">announced the latest additions, amendments, and deletions</a> from the Pharmaceutical Benefits Scheme (PBS): the program through which essential medicines are subsidised for Australian patients. The new medicines on the scheme are reportedly worth <a href="http://www.greghunt.com.au/Media/MediaReleases/tabid/86/ID/4230/Making-310-million-of-new-vital-drugs-available-for-Australian-patients.aspx">A$310 million</a>.</p>
<p>Listing on the PBS is different to a drug being approved for sale by Australia’s drug regulator, the <a href="http://www.tga.gov.au">Therapeutic Goods Administration (TGA)</a>. Once approved by the TGA, it is available to patients and hospitals at the full price. It only becomes subsidised if later listed on the PBS. </p>
<p>Some of the notable additions to the list include drugs to treat eye infections, human immunodeficiency virus (HIV), cystic fibrosis, multiple sclerosis, cancer, and <a href="http://lungfoundation.com.au/wp-content/uploads/2012/06/Idiopathic-Pulmonary-Fibrosis.pdf">idiopathic pulmonary fibrosis</a> – a type of scarring in the lungs. Below is a list of seven most notable new additions to the scheme.</p>
<h2>1. Chloramphenicol eye drops (Chlorsig)</h2>
<ul>
<li><p><strong>Maximum cost to Aboriginal and Torres Strait Islanders: A$0-6.10</strong></p></li>
<li><p><strong>Maximum cost to other patients: A$20.11</strong></p></li>
</ul>
<p>Chloramphenicol is the generic name of an antibiotic drug used to treat eye infections. It has been <a href="http://www.pbs.gov.au/medicine/item/11112W">added to the PBS</a> with the restriction that it is only available to patients who identify as Aboriginal or Torres Strait Islander (ATSI). </p>
<p>The rate of eye infections, like <a href="https://theconversation.com/why-is-trachoma-blinding-aboriginal-children-when-mainstream-australia-eliminated-it-100-years-ago-63526">trachoma</a> (which leads to blindness), is three times higher for ATSI patients than for other Australians. The lower price for ATSI patients is because of extra funding under the government’s <a href="http://www.pbs.gov.au/info/publication/factsheets/closing-the-gap-pbs-co-payment-measure">Closing the Gap PBS co-payment</a> program.</p>
<h2>2. Ivacaftor (Kalydeco)</h2>
<ul>
<li><p><strong>Maximum price from the manufacturer: A$22,547.02</strong></p></li>
<li><p><strong>Maximum cost to the patient: A$38.80</strong></p></li>
</ul>
<p>Ivacaftor – <a href="https://www.ebs.tga.gov.au/servlet/xmlmillr6?dbid=ebs/PublicHTML/pdfStore.nsf&docid=E059E457A00C1B93CA2580D0003CA6C0&agid=(PrintDetailsPublic)&actionid=1">first approved by the TGA in September 2016</a> – is used to treat <a href="http://www.cysticfibrosis.org.au/all/learn/">cystic fibrosis</a>, a genetic disorder that affects the digestive system and lungs of patients. It causes a buildup of thick and sticky mucus in the airways. </p>
<p>There is no cure for cystis fibrosis, but <a href="http://www.kalydeco.com/">ivacaftor</a> acts by better regulating the flow of salts and water in and out of cells, which leads to less mucus buildup. </p>
<p>While <a href="https://www.cysticfibrosis.org.au/media/wysiwyg/CF-Australia/medical-documents/CFA_DataRegistryReport_2014_Final.pdf">around 3,300 people in Australia live with cystic fibrosis</a>, only around 10% of patients will benefit from the drug. This is because patients need to have a specific mutation in their DNA called <em>R117H</em> for the drug to be effective. </p>
<hr>
<p><em><strong>More information - <a href="https://theconversation.com/weekly-dose-kalydeco-the-drug-that-treats-the-cause-of-cystic-fibrosis-not-just-symptoms-76934">Weekly Dose: Kalydeco, the drug that treats the cause of cystic fibrosis, not just symptoms</a></strong></em></p>
<hr>
<h2>3. Blinatumomab (Blincyto)</h2>
<ul>
<li><p><strong>Maximum price from the manufacturer: A$61,975.54</strong></p></li>
<li><p><strong>Maximum cost to the patient: A$38.80</strong></p></li>
</ul>
<p><a href="http://www.pbs.gov.au/medicine/item/11115B-11116C-11117D-11118E-11119F-11120G">Blinatumomab</a> is a new type of immunotherapy – a treatment that <a href="https://theconversation.com/au/search?utf8=%E2%9C%93&q=immunotherapy+">empowers the body’s immune system</a> to fight diseases such as cancer.</p>
<p>The drug is approved for use to treat a specific subset of <a href="http://www.leukaemia.org.au/blood-cancers/leukaemias/acute-lymphoblastic-leukaemia-all">acute lymphoblastic leukaemias</a> (ALL). Around 350 Australians each year are diagnosed with some form of ALL, and it is the most common type of cancer in children.</p>
<p>Blinatumomab was first approved by the TGA in November 2015 but an application to list the medicine on the PBS that same year <a href="http://www.pbs.gov.au/industry/listing/elements/pbac-meetings/psd/2015-11/files/blinatumomab-psd-november-2015.pdf">was rejected</a>. It <a href="https://www.greghunt.com.au/Home/LatestNews/tabid/133/ID/4230/Making-310-million-of-new-vital-drugs-available-for-Australian-patients.aspx">has been reported</a> that the cost for patients before the PBS subsidy was A$127,700 per course of treatment. </p>
<h2>4. Fosaprepitant (Emend IV)</h2>
<ul>
<li><p><strong>Maximum price from the manufacturer: A$115.03</strong></p></li>
<li><p><strong>Maximum cost to the patient: A$38.80</strong></p></li>
</ul>
<p>This drug is used to help patients overcome the nausea and vomiting side-effects
associated with chemotherapy treatment. <a href="http://www.pbs.gov.au/medicine/item/11103J-11107N">Fosaprepitant</a> has been available for doctors to prescribe since 2011, when it was <a href="https://www.pbs.gov.au/pbs/industry/listing/elements/pbac-meetings/pbac-outcomes/2011-03/positive-recommendations">first recommended</a> to be put on the PBS.</p>
<h2>5. Emtricitabine</h2>
<ul>
<li><p><strong>Maximum price from the manufacturer: A$1,500 - $2,600 depending on the formulation</strong></p></li>
<li><p><strong>Maximum cost to the patient: A$38.80</strong></p></li>
</ul>
<p>Four <a href="http://www.pbs.gov.au/pbs/search?term=EMTRICITABINE&analyse=false&search-type=medicines">formulations of this drug</a> have been added to the PBS as part of a cocktail of medicines used to treat HIV infection. </p>
<p>Emtricitabine acts by stopping the HIV virus from copying itself into human cells. It was first <a href="https://www.ebs.tga.gov.au/servlet/xmlmillr6?dbid=ebs/PublicHTML/pdfStore.nsf&docid=5C59BB9C228068B0CA257FE8004216C7&agid=(PrintDetailsPublic)&actionid=1">approved by the TGA in 2005</a> and other formulations of the drug – such as it being <a href="http://www.pbs.gov.au/medicine/item/10347N">coupled with antiviral Tenofovir</a> under the brand name Truvada – have been listed on the PBS previously. In Australia, there are around <a href="http://www.hivmediaguide.org.au/hiv-in-australia/hiv-statistics-australia/">25,000 people living with HIV</a>.</p>
<hr>
<p><em><strong>More information: <a href="https://theconversation.com/weekly-dose-truvada-the-drug-that-can-prevent-hiv-infection-61525">Weekly Dose: Truvada, the drug that can prevent HIV infection</a></strong></em></p>
<hr>
<h2>6. Daclizumab (Zinbryta)</h2>
<ul>
<li><p><strong>Maximum price from the manufacturer: A$2,231</strong></p></li>
<li><p><strong>Maximum cost to the patient: A$38.80</strong></p></li>
</ul>
<p><a href="http://www.pbs.gov.au/medicine/item/11101G">Daclizumab</a> is used to treat <a href="https://www.msaustralia.org.au/what-ms">multiple sclerosis (MS)</a>, a condition that affects the nervous system and interferes with nerve impulses in the brain, spinal chord, and optic nerves (those responsible for vision). It was first <a href="https://www.ebs.tga.gov.au/servlet/xmlmillr6?dbid=ebs/PublicHTML/pdfStore.nsf&docid=CC481F65EA7BE336CA258060003CA91D&agid=(PrintDetailsPublic)&actionid=1">approved by the TGA in September 2016</a>. </p>
<p>While there is no cure for MS, this drug helps to stop infection-fighting blood cells called <a href="http://www.medicinenet.com/script/main/art.asp?articlekey=11300">T-cells</a> from getting into the brain. This protects the brain from swelling. There are currently around 24,000 Australians who live with MS.</p>
<h2>7. Nintedanib (Ofev)</h2>
<ul>
<li><p><strong>Maximum price from the manufacturer: A$3,385.48</strong></p></li>
<li><p><strong>Maximum cost to the patient: A$38.80</strong></p></li>
</ul>
<p><a href="http://www.pbs.gov.au/medicine/item/11100F-11106M">Nintedanib</a> was <a href="https://www.ebs.tga.gov.au/servlet/xmlmillr6?dbid=ebs/PublicHTML/pdfStore.nsf&docid=0AEB4B2429304039CA2580B2003CA221&agid=(PrintDetailsPublic)&actionid=1">approved by the TGA in September 2015</a>. It is used to treat <a href="http://lungfoundation.com.au/wp-content/uploads/2012/06/Idiopathic-Pulmonary-Fibrosis.pdf">idiopathic pulmonary fibrosis</a>, a condition that causes scarring in the lungs. The amount of scar disease builds up over time. While nintedanib does not cure patients, it provides relief by stopping the enzymes that help create the scarring, thus slowing the disease.</p>
<p>The condition is most prevalent in people over 60 years of age, and <a href="http://lungfoundation.com.au/wp-content/uploads/2012/06/Idiopathic-Pulmonary-Fibrosis.pdf">affects around 2,600 Australians</a>.</p><img src="https://counter.theconversation.com/content/77090/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Dr Wheate in the past has received funding from the ACT Cancer Council, Tenovus Scotland, Medical Research Scotland, Scottish Crucible, and the Scottish Universities Life Sciences Alliance. He is affiliated with the Royal Australian Chemical Institute.</span></em></p>An independent expert provides his pick of the most notable drugs added to the PBS on May 1, 2017.Nial Wheate, Senior Lecturer in Pharmaceutics, University of SydneyLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/742812017-03-17T18:15:15Z2017-03-17T18:15:15ZHow big data is being mobilised in the fight against leukaemia<figure><img src="https://images.theconversation.com/files/161168/original/image-20170316-10892-11x4lcj.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Leukaemia: information may be the best cure.</span> <span class="attribution"><a class="source" href="https://www.shutterstock.com/image-illustration/leukaemia-white-blood-cells-3d-illustration-536066266?src=6-O8Oxyp9a2t0uQfCOaMgA-1-2">Shutterstock</a></span></figcaption></figure><p>Healthy cell function relies on well orchestrated gene activity. Via a fantastically complex network of interactions, around 30,000 genes cooperate to maintain this delicate balance in each of the <a href="http://www.smithsonianmag.com/smart-news/there-are-372-trillion-cells-in-your-body-4941473/">37.2 trillion cells</a> in the human body. </p>
<p>Broadly speaking, cancer is a disruption of this balance by genetic changes, or mutations. Mutations can trigger over-activation of genes that normally instruct cells to divide, or inactivation of genes that suppress the development of cancer. When a mutated cell divides, it passes the mutation down to its daughter cells. This leads to the accumulation of non-functioning, abnormal cells that we recognise as cancer.</p>
<p><a href="http://www.gla.ac.uk/researchinstitutes/cancersciences/staff/tessaholyoake/">Our laboratory</a> is focused on understanding how one particular cancer – <a href="http://www.nhs.uk/Conditions/Leukaemia-chronic/Pages/Introduction.aspx">chronic myeloid leukaemia</a> or CML – works. Each year more than 700 patients in the UK – and <a href="http://www.who.int/selection_medicines/committees/expert/20/applications/CML.pdf?ua=1">over 100,000</a> worldwide – are diagnosed with CML. After recent advances, <a href="http://www.cancerresearchuk.org/about-cancer/chronic-myeloid-leukaemia-cml/survival">almost 90%</a> of patients under the age of 65 now survive for more than five years. </p>
<p>But in the vast majority of patients CML is currently incurable and lifelong treatment means that patients must live with side effects and the chance of drug resistance arising. With increasing numbers of CML patients surviving (and treatment costing between £40,000 and £70,000 per patient a year), increasing strain is being placed on health services. </p>
<h2>A single mutation</h2>
<p>CML is perhaps unique in cancers in that a single mutation, named <a href="http://www.sciencedirect.com/science/article/pii/S0950353697800029">BCR-ABL</a>, underlies the disease biology. This mutation originates in a single <a href="http://www.bloodjournal.org/content/94/6/2056.long">leukaemic stem cell</a>, but is then propagated throughout the blood and bone marrow as leukaemia cells take over and block the healthy process of blood production. The presence of BCR-ABL affects the activity of thousands of genes, in turn preventing these cells from fulfilling their normal function as blood cells.</p>
<p><a href="http://www.nature.com/nm/journal/v2/n5/abs/nm0596-561.html">Drugs</a> that specifically neutralise the aberrant effects of this mutation were introduced to the clinic from the early 2000s. These drugs have revolutionised CML patient care. Many are now able to live relatively normal lives with their leukaemia under good control. </p>
<p>But while these drugs kill the more mature daughter cells of the originally mutated leukaemia stem cell, they have not fully lived up to their initial billing as “magic bullets” in the fight against cancer. This is because the original “seed” population of leukaemic stem cells <a href="http://www.bloodjournal.org/content/119/6/1501?sso-checked=true">evade therapy</a>, <a href="http://www.bloodjournal.org/content/118/20/5565.long?sso-checked=true">lying dormant in the bone marrow</a> to stimulate new cancer growth when treatment is withdrawn. </p>
<figure class="align-center ">
<img alt="" src="https://images.theconversation.com/files/161164/original/image-20170316-10902-ttzpo8.JPG?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/161164/original/image-20170316-10902-ttzpo8.JPG?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=450&fit=crop&dpr=1 600w, https://images.theconversation.com/files/161164/original/image-20170316-10902-ttzpo8.JPG?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=450&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/161164/original/image-20170316-10902-ttzpo8.JPG?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=450&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/161164/original/image-20170316-10902-ttzpo8.JPG?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=565&fit=crop&dpr=1 754w, https://images.theconversation.com/files/161164/original/image-20170316-10902-ttzpo8.JPG?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=565&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/161164/original/image-20170316-10902-ttzpo8.JPG?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=565&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">Chronic Myeloid Leukemia smear.</span>
<span class="attribution"><a class="source" href="https://commons.wikimedia.org/w/index.php?title=Special:Search&title=Special:Search&redirs=0&search=chronic+myeloid+leukaemia&fulltext=Search&fulltext=Advanced+search&ns0=1&ns6=1&ns14=1&advanced=1&searchToken=9mbmkxv3su58o9lrng2fa2f34#/media/File:Chronic_Myeloid_Leukemia_smear_2009-04-09.JPG">Paulo Henrique Orlandi Mourao via Wikimedia Commons.</a>, <a class="license" href="http://creativecommons.org/licenses/by-sa/4.0/">CC BY-SA</a></span>
</figcaption>
</figure>
<p>To truly cure CML we must expose, understand the inner workings of, and uproot the leukaemia stem cells. And to do this, we need to learn more about them. How do they survive the treatment that so readily kills their more mature counterparts? Which overactive or inactivated genes protect them?</p>
<p>We believe that the answers to these questions lie in the analysis of biological “big data”. Genome-scale technologies now allow scientists to measure the activity (or “expression”) of every gene in the genome simultaneously, in any given population of cells, or even at the level of a single cell. Comparison of expression data generated from leukaemia stem cells with the same data generated from healthy blood stem cells will reveal single genes or networks of genes potentially targetable in the fight against leukaemia.</p>
<h2>Big data to the rescue</h2>
<p>In a project funded by Bloodwise and the Scottish Cancer Foundation, we have created <a href="http://www.stemformatics.org/leukomics">LEUKomics</a>. This online data portal brings together a wealth of CML gene expression data from specialised laboratories across the globe, including our own at the University of Glasgow. </p>
<p>Our intention is to eliminate the bottleneck surrounding big data analysis in CML. Each dataset is subjected to manual quality checks, and all the necessary <a href="http://www.sciencedirect.com/science/article/pii/S1873506112001262">computational processing</a> to extract information on gene expression. This enables immediate access to and interpretation of data that previously would not have been easily accessible to academics or clinicians without training in specialised computational approaches.</p>
<p>Consolidating these data into a single resource also allows large-scale, computationally-intensive research efforts by bioinformaticians (specialists in the analysis of big data in biology). From a computational perspective, the fact that CML is caused by a single mutation makes it an attractive disease model for cancer stem cells. However, existing datasets tend to have small sample numbers, which can limit their potential. </p>
<p>The more samples available, the higher the power to detect subtle changes that may be crucial to the biology of the cancer stem cells. By bringing all the globally available CML datasets together, we have significantly increased the sample size, from two to six per dataset to more than 100 altogether. This offers an unprecedented opportunity to analyse gene expression data to expose underlying mechanisms of this disease.</p>
<p>As of March 2017, the <a href="http://www.stemformatics.org/leukomics">portal</a> is up and running in the public domain. We are planning to tour Scotland and present at international conferences, aiming to train researchers in how best to exploit this new resource. Ultimately, we hope that this tool will lead to new ideas and approaches, and attract more funding, in the fight against CML. And while we continue to expand our representation of CML data in real time from research centres all over the world, we also plan to begin incorporating data from other types of leukaemia.</p>
<p>In recent years, targeted therapies have become <a href="https://theconversation.com/learning-the-language-of-cells-will-help-scientists-beat-cancer-58528">hugely important</a> in cancer research. By providing these data to the CML research community within <a href="http://www.stemformatics.org/leukomics">LEUKomics</a>, we hope to mobilise new research into cancer-causing leukaemic stem cells, and ultimately design treatments to target them without affecting healthy cells. Our database provides a critical stepping stone in this process.</p><img src="https://counter.theconversation.com/content/74281/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Lorna Jackson receives funding from the Medical Research Council, the Scottish Cancer Foundation and Bloodwise. </span></em></p><p class="fine-print"><em><span>Lisa Hopcroft has received funding from Cancer Research UK and Bloodwise. </span></em></p>A new data portal bypasses the big data bottleneck to find targeted therapies for leukaemia.Lorna Jackson, PhD candidate (Paul O'Gorman Leukaemia Research Centre), University of GlasgowLisa Hopcroft, Research Associate (Institute of Cancer Sciences), University of GlasgowLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/616662016-07-31T20:07:33Z2016-07-31T20:07:33ZChildhood cancer deaths have fallen in Australia, but some types remain more of a challenge<figure><img src="https://images.theconversation.com/files/132133/original/image-20160727-5663-143ky4o.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Death rates from childhood cancer in Australia have fallen sharply.</span> <span class="attribution"><span class="source">from shutterstock.com</span></span></figcaption></figure><p>Childhood deaths from cancer have decreased by nearly 40% in the past 15 years in Australia, our <a href="http://www.anr2016.org/anzchog/full-program/">latest figures show</a>. While overall cancer rates have increased during the past 30 years, mortality rates have dropped, both overall and for particular types of childhood cancer.</p>
<p>According to unpublished data from the <a href="https://cancerqld.org.au/research/cancer-registries/australian-paediatric-cancer-registry/">Australian Paediatric Cancer Registry</a>, childhood cancer death rates decreased by about 3% per year between 1998 and 2013. There have been significant improvements in survival for childhood leukaemias, lymphomas, neuroblastoma and malignant bone tumours.</p>
<p>Today, tumours of the central nervous system (mainly brain tumours) account for 40% of all childhood cancer deaths, the single largest cause of cancer deaths for children in Australia. These are followed by leukaemias (23%) and <a href="http://www.neuroblastoma.org.au/about-neuroblastoma/overview/">neuroblastoma</a> (12%), which is the most common solid tumour in childhood, usually found in the abdomen.</p>
<p>Death rates from leukaemia – the most common form of childhood cancer – have dropped from 16.6 deaths per million children in 1998 to 4.4 in 2013. This equates to an overall decrease of 68% over that period. </p>
<iframe src="https://datawrapper.dwcdn.net/25agA/1/" frameborder="0" allowtransparency="true" allowfullscreen="allowfullscreen" webkitallowfullscreen="webkitallowfullscreen" mozallowfullscreen="mozallowfullscreen" oallowfullscreen="oallowfullscreen" msallowfullscreen="msallowfullscreen" width="100%" height="400"></iframe>
<p>Five-year survival for all childhood cancers increased from 77% for the period 1994-2003 to 84% for 2004-2013. We know 98% of children who survive the first five years after diagnosis will survive a further five years.</p>
<p>However, there was little or no survival change for several types of childhood cancers over recent years. These included brain, kidney and liver cancers. </p>
<h2>Childhood cancer in Australia</h2>
<p>Australia has the <a href="https://cancerqld.org.au/news/australian-childhood-cancer-deaths-drop-sharply/">lowest childhood cancer death</a> rate of all G20 countries. And Australia’s five-year survival rates for childhood cancer are among the best in the world. </p>
<p>The key reason for the downward trends of death rates is improved treatment, particularly the introduction of more effective chemotherapy protocols and adjunct therapies such as radiotherapy. Cancer research, especially clinical trials, has contributed to improved treatments and better survival rates.</p>
<p>However, our figures also show Australia has one of the highest childhood cancer rates in the world. Incidence of childhood cancer increased significantly from 1983 until the mid-1990s, but has remained fairly constant since. </p>
<iframe src="https://datawrapper.dwcdn.net/tQJdA/4/" frameborder="0" allowtransparency="true" allowfullscreen="allowfullscreen" webkitallowfullscreen="webkitallowfullscreen" mozallowfullscreen="mozallowfullscreen" oallowfullscreen="oallowfullscreen" msallowfullscreen="msallowfullscreen" width="100%" height="400"></iframe>
<p>Cancer is the second leading cause of death in Australian children, behind injury and poisoning. Around 710 children aged under 15 are diagnosed with cancer each year and about 100 die from the disease. </p>
<p>The causes of most childhood cancers and factors driving changes in incidence over time are largely unknown. Unlike cancer in adults, where <a href="https://theconversation.com/interactive-body-map-what-really-gives-you-cancer-52427">behavioural factors</a> such as smoking, alcohol consumption, diet and exercise play an important role, almost nothing is known about what may increase cancer risk in children. </p>
<p>A possible exception to this is melanoma. Our figures show a 38% decrease in the incidence of melanoma among children in Australia between 1993 and 2013.
This is most likely the result of long-running public health campaigns such as Slip, Slop, Slap that promote improved sun protection, particularly among children.</p>
<h2>Tough cancers in children</h2>
<p>In the most recent period, almost all children (98%) diagnosed with the rare eye-cancer <a href="https://childrenscancer.canceraustralia.gov.au/types-childrens-cancers/retinoblastoma">retinoblastoma</a> survived for at least five years. Five-year survival rates also exceeded 90% for lymphomas (cancers of the lymphatic system), germ cell tumours (most of which are occur in the ovaries and testes) and the group of cancers that includes melanoma. </p>
<p>In contrast, five-year survival was more moderate (between 70% to 75%) for children with tumours of the liver, the central nervous system (mainly brain tumours) and neuroblastoma. Five-year survival for tumours of the central nervous system has only slightly improved from 71% in 1994-2003 to 74% in 2004-2013.</p>
<p>The incidence of liver cancer in children is increasing by 2.5% per year, compared to a stable trend of 0.2% per year increase in the rate for all childhood cancers combined.</p>
<figure class="align-center ">
<img alt="" src="https://images.theconversation.com/files/132143/original/image-20160727-5638-2qgiki.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&fit=clip" srcset="https://images.theconversation.com/files/132143/original/image-20160727-5638-2qgiki.png?ixlib=rb-1.1.0&q=45&auto=format&w=600&h=411&fit=crop&dpr=1 600w, https://images.theconversation.com/files/132143/original/image-20160727-5638-2qgiki.png?ixlib=rb-1.1.0&q=30&auto=format&w=600&h=411&fit=crop&dpr=2 1200w, https://images.theconversation.com/files/132143/original/image-20160727-5638-2qgiki.png?ixlib=rb-1.1.0&q=15&auto=format&w=600&h=411&fit=crop&dpr=3 1800w, https://images.theconversation.com/files/132143/original/image-20160727-5638-2qgiki.png?ixlib=rb-1.1.0&q=45&auto=format&w=754&h=517&fit=crop&dpr=1 754w, https://images.theconversation.com/files/132143/original/image-20160727-5638-2qgiki.png?ixlib=rb-1.1.0&q=30&auto=format&w=754&h=517&fit=crop&dpr=2 1508w, https://images.theconversation.com/files/132143/original/image-20160727-5638-2qgiki.png?ixlib=rb-1.1.0&q=15&auto=format&w=754&h=517&fit=crop&dpr=3 2262w" sizes="(min-width: 1466px) 754px, (max-width: 599px) 100vw, (min-width: 600px) 600px, 237px">
<figcaption>
<span class="caption">Long-running public health campaigns such as Slip Slop Slap likely contributed to decreased incidence of melanoma in Australian children.</span>
<span class="attribution"><a class="source" href="http://robotzo.blogspot.com.au/2011/10/boc-slip-slap-slop.html">Screenshot Robotzo Blogspot</a></span>
</figcaption>
</figure>
<p>The causes of liver cancer in children are largely unknown. Hepatoblastoma, a type of liver cancer that occurs in children aged under four, can occur in association with certain genetic syndromes such as <a href="https://ghr.nlm.nih.gov/condition/familial-adenomatous-polyposis">familial adenomatous polyposis</a> and <a href="https://ghr.nlm.nih.gov/condition/beckwith-wiedemann-syndrome">Beckwith-Wiedemann syndrome</a>, also known as overgrown syndrome. It has also <a href="http://www.so-online.net/article/S0960-7404(07)00068-0/abstract">occurred more frequently among infants</a> delivered prematurely.</p>
<p>The risk of hepatic carcinoma, a type of liver cancer mainly affecting children aged over ten, appears to increase among those who have had other liver diseases such as cirrhosis, metabolic diseases or chronic hepatitis. </p>
<p>The reasons for the apparent increase in the incidence of liver cancer are unknown and an international study involving the Australia Paediatric Cancer Registry is being planned to examine issues around childhood liver cancer.</p>
<p>Children who survive cancer can suffer serious long-term consequences resulting from their treatment. These <a href="http://www.cancer.org/treatment/childrenandcancer/whenyourchildhascancer/children-diagnosed-with-cancer-late-effects-of-cancer-treatment">late effects are many and varied</a> and depend on factors such as the type of cancer and the treatment the child received.</p>
<p>Some examples include <a href="https://theconversation.com/many-survivors-of-childhood-brain-cancer-have-cognitive-difficulties-but-these-can-be-treated-57566">learning and behavioural problems</a>, vision and hearing deficiencies, stunted growth, heart disease, reduced lung capacity, dental issues, impaired sexual development and a higher risk of second cancers.</p>
<p>As well as improving survival, it’s important to find better therapies that do not impact on long-term health and quality of life.</p><img src="https://counter.theconversation.com/content/61666/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Joanne Aitken receives funding from Cancer Council Queensland and Cancer Australia. </span></em></p>Childhood deaths from cancer have decreased by nearly 40% in the past 15 years in Australia. But some types of childhood cancer have shown little improvement.Joanne Aitken, Professor and Head of Research, Cancer Council QueenslandLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/599142016-05-25T10:59:06Z2016-05-25T10:59:06ZRevisiting Frantz Fanon: memories and moments of a militant philosopher<figure><img src="https://images.theconversation.com/files/123795/original/image-20160524-25209-1vb60kr.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Twentieth-century political thinker and fighter against colonialism and imperialism, Frantz Fanon, left an indelible mark on history.</span> <span class="attribution"><span class="source">Tony Webster/Flickr</span></span></figcaption></figure><p><em>A new <a href="http://www.ibtauris.com/Books/Humanities/Philosophy/Social%20%20political%20philosophy/Frantz%20Fanon%20The%20Militant%20Philosopher%20of%20Third%20World%20Liberation.aspx?menuitem=%7B65A3FB7C-5D2E-4158-BBA9-D7824186AD5B%7D">book</a> by Leo Zeilig focuses on one of Africa and the modern era’s most celebrated revolutionaries, whose views remain influential long after his death. This is an edited extract.</em></p>
<p>Many people spoke well that day. Delegates had come from across the African continent to independent Ghana for the <a href="http://africanactivist.msu.edu/document_metadata.php?objectid=32-130-D84">All-African Peoples’ Conference</a> in 1958. Most spoke of the continuing struggle against colonialism. </p>
<p>In the Congo, labelled the “empire of silence”, the <a href="http://www.gutenberg.us/articles/mouvement_national_congolais-lumumba">Mouvement National Congolais</a> faced repression by a colonial power that refused to entertain any notion of genuine independence. </p>
<p>In South Africa, the apartheid regime was confident that it could keep at bay the increasing demands for change north of its borders. President Charles de Gaulle had offered limited sovereignty to French colonies. Later that year Guinea, under Ahmed Sékou Touré, would insist on immediate independence:</p>
<blockquote>
<p>We prefer freedom in poverty to riches in slavery.</p>
</blockquote>
<p>In colonies where there was a large white “settler” presence, the struggle against colonial rule was deeper and more protracted. In others, the colonial metropolis had begun to accept the inevitability of decolonisation. As the delegates gathered that day, only Ghana had become independent.</p>
<p><a href="https://www.modernghana.com/lifestyle/1094/16/biography-of-dr-kwame-nkrumah.html">Kwame Nkrumah</a> led a black government, speaking openly of breaking the chains of colonialism and imperialism, and of pan-African solidarity and socialism. After generations of slavery, colonialism and racism, here was a country that seemed to declare to the world what a victorious and united movement of liberation could achieve. Though Nkrumah had not defeated colonial armies militarily, like <a href="http://www.blackpast.org/gah/loverture-toussaint-1742-1803">Toussaint Louverture</a> had in Haiti, Ghana stood proud and defiant in a world still dominated by racism.</p>
<p><a href="http://www.ibtauris.com/Books/Humanities/Philosophy/Social%20%20political%20philosophy/Frantz%20Fanon%20The%20Militant%20Philosopher%20of%20Third%20World%20Liberation.aspx?menuitem=%7B65A3FB7C-5D2E-4158-BBA9-D7824186AD5B%7D">Frantz Fanon</a> could not help himself. This intense, direct, Caribbean-born doctor and revolutionary would soon have a reputation for capturing the world’s anger. When he mounted the podium to speak, most delegates did not know who he was, nor had they read any of his writings. His eyes, fixed on his text, shone with urgency and intensity. </p>
<blockquote>
<p>If Africa is to be free, we cannot beg. We must tear away by force.</p>
</blockquote>
<p>All forms of struggle must be adopted, he argued, not excluding violence. The delegates were transfixed. The South African writer <a href="http://www.sahistory.org.za/people/dr-eskia-mphahlele">Es'kia Mphahlele</a> wrote:</p>
<blockquote>
<p>Dr Fanoh Omar [sic] of Algeria is certainly the highlight of the session. He does not mince words, what FLN [Algeria’s Front de la Liberation Nationale] man can afford the luxury anyway? Algerians have no other recourse but fight back, he says, and the FLN means to go through with it. In staccato French he carries the audience to the horrible scene of French atrocities on Algerians … He gets the loudest and longest ovation of all the speakers. </p>
</blockquote>
<p>For Fanon it was not enough to celebrate the achievements of decolonisation. It was necessary to educate, to strain at the limits of national freedom and provoke debate. The All-African Peoples’ Conference was the place to do this, and to learn about other movements on the continent. </p>
<p>Only two years later, in 1960, he would represent the Algerian <a href="http://countrystudies.us/algeria/33.htm">provisional government</a> (Gouvernement Provisoire de la République Algérienne) in independent Ghana, less than six years after he joined the Algerian struggle. Ghana was both a headquarters for independence movements and a laboratory for actually existing independent nationhood. </p>
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<p>It was already a collection of vivid and painful contradictions. Many white people had stayed on to assist the new government. Even the army was temporarily being run by British officers. But Nkrumah was an outspoken advocate for <a href="https://theconversation.com/sobukwes-pan-africanist-dream-an-elusive-idea-that-refuses-to-die-52601">pan-Africanism</a>. For a generation of young militants he was a figure to emulate. Fanon learned much in Ghana.</p>
<p>In 1961, his life ebbing away from leukaemia, Fanon dictated his masterwork, “The Wretched of the Earth”, to his wife, friends and secretaries. Finding some strength after a new round of treatment, he travelled to the Tunisian/Algerian border (Ghardimaou in Tunisia) and spoke to the <a href="http://global.britannica.com/topic/National-Liberation-Front-political-party-Algeria">Armée de Libération Nationale</a> as it prepared to fight the French and enter a free Algeria.</p>
<p>In this last public appearance, he read to the assembled troops, many of them illiterate, from his draft of what would become the most famous chapter in “<a href="http://home.ku.edu.tr/%7Embaker/CSHS503/FrantzFanon.pdf">Wretched of the Earth</a>”, about the pitfalls of national consciousness.</p>
<p>He described how the national bourgeoisie, after independence, is only too happy to accept what crumbs the departing colonial powers throw to it. Without social reform, without political and economic transformation, he warned, national liberation would be an empty shell. </p>
<p>Fanon’s final act was to encourage – and yet subvert – the revolutionary movement to which he had devoted the last and most important years of his life. He had stubbornly refused to accept treatment in the United States, which he condemned for its racism. </p>
<p>But, in October 1961, after this final and exhausting resurrection, he flew there from Tunisia, his home in exile. His last Atlantic crossing was to no avail. On December 6 1961 he died at 36 years of age. Since his death Fanon has been endlessly resurrected, sometimes bastardised, often
deified. </p>
<p>In his adoptive Algeria, which won independence in 1962 after a gruelling eight-year war that killed hundreds of thousands of Algerians, he has received uneasy recognition. His work has been translated into Arabic, his old hospital in Blida named after him, a school and large street carry his name in Algiers. </p>
<p>But his warnings were grimly fulfilled. In the mid-1960s a new <a href="http://hoover.archives.gov/exhibits/africanamerican/blackpower/">Black Power movement</a>, principally in the United States, took up Fanon’s writings. It interpreted his analysis of racism and his insistence on the necessity of organising the wretched of the earth, and on the therapeutic effects of violence as defence against oppression, as tools to deploy against the “colonisation” of black communities there.</p>
<p>Bobby Seale, co-founder of the Black Panther Party, cited the influence of </p>
<blockquote>
<p>everything that Fanon said about violence and the spontaneity of violence, how spontaneous violence educates those who are in a position with skills to lead the people to what needs to be done.</p>
</blockquote>
<p>Others who claimed to understand Fanon’s legacy were not so generous or hopeful. Critics and fellow travellers alike declared him a prophet of violent revolution, accusing him of championing the detoxifying and cleansing effects of violence without appreciating its destructive and degenerative whirlwind. </p>
<p>In the 1980s and 1990s, renewed interest in Fanon painted him as a scholar and theorist of identity, masculinity, postcolonialism and subaltern studies. Some of these labels may be justified, but they are also misleading.</p>
<p>The academy’s adoption of radical thinkers is always a sanitising process, turning revolutionary action into passive reflection, analysis into academic pontification. To read Fanon in the 1980s was to cherry pick from a postmodern orchard, divorcing his work on racism, subjectivity and lived experience from its wider revolutionary context and its untidy dialectic.</p>
<p>For others, Fanon – the insistent revolutionary, arguing for education, analysis and practice – became a romantic myth-monger of the Algerian revolution, positing liberation when none was on offer. Yet his philosophical work and interests were always contingent on action and contributing to real practice. </p>
<p>It would be churlish to dismiss this re-engagement with Fanon: his work was extraordinarily complex and its insights extended beyond disciplinary boundaries to include psychiatry, philosophy and politics. For more than 50 years dozens of important biographies, collections and studies have emerged on his work and life.</p>
<p><em>“<a href="http://www.ibtauris.com/Books/Humanities/Philosophy/Social%20%20political%20philosophy/Frantz%20Fanon%20The%20Militant%20Philosopher%20of%20Third%20World%20Liberation.aspx?menuitem=%7B65A3FB7C-5D2E-4158-BBA9-D7824186AD5B%7D">Frantz Fanon: Militant Philosopher of the Third World Revolution</a>” is published by <a href="http://www.ibtauris.com/">IB Tauris</a>.</em></p><img src="https://counter.theconversation.com/content/59914/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Leo Zeilig 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>For the revolutionary Frantz Fanon it was not enough to celebrate the achievements of decolonisation. It was necessary to educate, to strain at the limits of national freedom and to provoke debate.Leo Zeilig, Senior Research Fellow, Institute of Commonwealth Studies, School of Advanced Study, University of LondonLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/496062015-11-11T11:17:44Z2015-11-11T11:17:44ZExplainer: what has been holding gene therapy back?<figure><img src="https://images.theconversation.com/files/101035/original/image-20151106-16253-1rzjd0s.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">The accuracy of gene editing will be the deciding factor in the success of current technology.</span> <span class="attribution"><a class="source" href="http://www.shutterstock.com">Genes by Shutterstock</a></span></figcaption></figure><p>Gene therapy is the ultimate comeback kid. A few weeks ago, it celebrated <a href="http://www.bsgct.org/en/education/a-history-of-gene-therapy.html">25 years</a> since the first trial in humans. Nobody cared. But when it was recently announced that a new type of gene therapy - only ever tested on mice - had been used to treat 17-month-old <a href="http://www.bbc.co.uk/news/health-34739304">Layla Richards’</a> leukaemia, everybody cared.</p>
<p>Given its huge potential, the passing of the anniversary with so little media coverage emphasises its stormy passage over the last quarter of a century. </p>
<h2>Replacing faulty genes</h2>
<p>Many human diseases, including cancer, are caused by defects that occur in the genes within our cells. Genes are the blueprints that allow cells to make the proteins that control how they function. A mutation to a gene can either change a protein so that it no longer works as it should or the protein is not made at all.</p>
<p>Gene therapy attempts to treat disease by correcting the defective gene or genes. It has been proposed as a treatment for a wide range of diseases including cancer, cystic fibrosis, haemophilia and Parkinson’s disease.</p>
<p>It is relatively easy to make a healthy gene in the laboratory. The problem is that the healthy gene needs to be delivered to the affected cells without causing problems for the patient. </p>
<p>Early gene therapy focused on replacing the patient’s defective gene with a healthy copy. This was achieved by using a harmless virus, a “viral vector”, to deliver the healthy genes to the patient’s cells. That was the idea, anyway. The big problem is targeting the gene to a safe location within the recipient cell. If it goes in the wrong place then the cell can be damaged.</p>
<h2>From glorious potential to disaster</h2>
<p>In 1999, the promise of using replacement genes to cure disease lay in tatters – just nine years after gene therapy began – after <a href="http://www.scientificamerican.com/article/gene-therapy-an-interview/">18-year-old volunteer</a> Jessie Gelsinger died from multiple organ failure following a trial at the University of Pennsylvania. The exact cause of the Gelsinger’s death is still a mystery, but the <a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC81135/">expert opinion</a> is that his immune system overreacted to the viral vector.</p>
<p>This tragic incident was a wake up call for scientists who had been caught up in the hype surrounding gene therapy and its perceived simplicity. </p>
<p>Further problems were to follow. In 2003, a French patient being treated for an immune system disorder, developed a <a href="http://www.nature.com/gt/journal/v10/n1/full/3301946a.html">leukaemia-like</a> condition as a direct result of the treatment. The viral vector mistakenly delivered a replacement gene into a healthy gene, called LMO-2, which <a href="http://www.ncbi.nlm.nih.gov/pubmed/12351380?dopt=Abstract">caused some cells to malfunction</a>. This gene <a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4632508/">has been linked</a> to a number of childhood cancers.</p>
<p>This disaster led to a moratorium on gene therapy trials. </p>
<h2>Renewed optimism</h2>
<p>Despite the setbacks, scientists and doctors have been slowly rebuilding the reputation of gene therapy and there is now a renewed optimism. Careful regulation of clinical trials involving gene therapy and improved gene technology that can silence overactive genes or repair – rather than replace them – has revitalised the field.</p>
<p>New gene editing technologies such as <a href="http://www.nature.com/mt/journal/v20/n9/full/mt2012171a.html">CRISPR</a> and <a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3547402/">TALEN</a> can be used to perform precision microsurgery on defective genes. This was demonstrated with the recent application of TALEN gene editing to treat <a href="http://www.bbc.co.uk/news/health-34739304">Layla Richards’</a> leukaemia at Great Ormond Street Hospital in London – the first time a gene editing technology had been used in a human. And the results appear to be almost miraculous. But, if history has taught us anything, it’s the need to be cautiously optimistic. </p>
<p>However, there is a real possibility that this particular case could be the breakthrough that gene therapy scientists and patients have been waiting for because Layla’s genes are being subtly altered rather than totally replaced. Ultimately, it will be the accuracy of these new gene editing technologies in targeting precise genetic alterations to a cell’s genes that will determine their future in the treatment of diseases such as cancer. </p>
<p>The targeting of genes within cells using this technology is certainly a huge advance on earlier techniques, with their random placement of genes and devastating consequences. However, there is evidence to suggest that changes to genes elsewhere in the cell – genes that aren’t the target of the therapy – does occur <a href="http://www.ncbi.nlm.nih.gov/pubmed/24837660">with these new editing methods</a>, but its hard to predict the frequency or say where and what the effects will be. In the end, it will be the extent of this deviation from the target gene site that will decide the technology’s fate and that of the patients undergoing treatment. </p>
<h2>Eye-watering cost</h2>
<p>Interest in gene therapy is going to increase in the next 10 to 20 years. One reason for this is the potential return on investment for biotechnology companies. A recently licensed European gene therapy treatment came with an eye-watering <a href="http://www.dailymail.co.uk/news/article-2226863/First-gene-therapy-drug-given-European-license.html">price tag of around €1m</a> per patient. </p>
<p>Other treatments currently in development have projected costs of around US$1m per patient. These record breaking treatment costs may be explained by the small number of patients diagnosed with the diseases for which the treatments were designed and the fact that gene therapy is typically thought of as a “once only” treatment.</p>
<p>The high costs of a particular gene therapy will likely persist until the patent protecting the invention expires or someone comes up with a different therapy for the same disease and the competition drives the price down.</p>
<p>However, biotech companies need to carefully consider how they price such technologies as extortionate fees will undoubtedly be fought by healthcare providers and patient-groups alike. Bad publicity surrounding prohibitively expensive treatments, especially for rare diseases in children, can be very damaging to a <a href="http://www.bbc.co.uk/news/world-us-canada-34331761">company’s reputation</a>. </p>
<p>As gene therapy looks forward to the next 25 years, there is a lot to be optimistic about, but unless the cost of treatment falls, gene therapy could remain as rare as the diseases it treats.</p><img src="https://counter.theconversation.com/content/49606/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>David Pye 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>A toddler from London has had gene therapy for leukaemia and it seems to have worked. But why have breakthroughs taken so long?David Pye, Scientific Director of the Kidscan Childrens Cancer Research Charity, University of SalfordLicensed as Creative Commons – attribution, no derivatives.tag:theconversation.com,2011:article/284782014-06-26T14:51:19Z2014-06-26T14:51:19ZCancer uses stem cells as a shield to escape drug attacks<figure><img src="https://images.theconversation.com/files/52363/original/m7xp3y95-1403793231.jpg?ixlib=rb-1.1.0&q=45&auto=format&w=496&fit=clip" /><figcaption><span class="caption">Cancer cell to stem cells: the drugs are coming.</span> <span class="attribution"><a class="source" href="https://www.flickr.com/photos/delgrossodotcom/3084576907">delgrossodotcom</a>, <a class="license" href="http://creativecommons.org/licenses/by-nc-sa/4.0/">CC BY-NC-SA</a></span></figcaption></figure><p>Chemotherapy is one of the most important treatments for all types of cancer. It involves the use of drugs that kill abnormally multiplying cells. The therapy uses one or more drugs in combination and has been practised since the 1950s, along with surgery and radiation therapy. </p>
<p>But chemotherapy does not always work. Doctors observe that even if the treatment is initially successful, patients often suffer a relapse. Sometimes this happens even after multiple sessions of chemotherapy. It seems some cancer cells manage to escape and hide within the body, allowing the disease to eventually return. </p>
<h2>A dance of proteins</h2>
<p>A recent study published in the journal <a href="http://dx.doi.org/10.1016/j.ccr.2014.04.015">Cancer Cell</a> throws light on how cancer cells manage to dodge chemotherapy. Deng-Li Hong, professor of pathophysiology at Jiao Tong University School of Medicine, and his colleagues discovered that some cancer cells kidnap the patient’s own stem cells.</p>
<p>With the onset of acute lymphoblastic leukaemia, Deng-Li and his associates found that some cancer cells hide in the bone marrow, where blood stem cells are located. Inside the bone marrow, their aim is to find some way to become resistant to cancer drugs, which they manage by the interaction of a series of proteins.</p>
<p>First, cancer cells give off signalling proteins called cytokines, which attract certain kinds of stem cells known as mesenchymal stem cells. After luring them, cancer cells form a nest within the stem cells and coax them to grow. </p>
<p>Cancer cells are aiming to harvest a special protein, called furin, from those mesenchymal stem cells. Furin has the unique ability to activate other proteins by chewing away useless parts of a protein, fine-tuning its structure and switching it on. Cancer cells use furin from the hostage stem cells to activate the master protein GDF15. GDF15 then activates the cancer cell’s defense system, making it resistant to chemotherapy. </p>
<h2>Without defence</h2>
<p>While the experiments were mostly performed in mice, Deng-Li found that the hostage situation also occurs in humans. Leukaemia patients who did not respond well to chemotherapy possess cancer cells surrounded by hostage cells. These cancer cells have already switched on GDF15 production, causing resistance to chemotherapy.</p>
<p>This doesn’t mean giving up on chemotherapy just yet. Some leukaemia patients respond well to chemotherapy and they don’t show any trace of the hostage situation.</p>
<p>Better understanding of how cancer cells resist drugs may help us find new weapons. If cancer cells depend on GDF15 to escape drugs, maybe drugs that also inhibit GDF15 could be added to the mix of drugs given in chemotherapy. </p>
<p>“This work gives us a handle on treatment regimes for patients of ALL,” Deng-Li said. “We could possibly use agents that prevent the hostage situation during the early stages of chemotherapy.”</p>
<p>GDF15 has been found to play a role in other types of cancers, such as pancreatic cancer and multiple myeloma. It could be part of the reason why chemotherapy doesn’t always work in these cancers as well. </p>
<hr>
<p><em>Next, read this: <a href="https://theconversation.com/now-we-know-why-drugs-dont-work-on-pancreatic-cancer-28420">Now we know why drugs don’t work on pancreatic cancer</a></em></p><img src="https://counter.theconversation.com/content/28478/count.gif" alt="The Conversation" width="1" height="1" />
<p class="fine-print"><em><span>Anwesha Ghosh 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>Chemotherapy is one of the most important treatments for all types of cancer. It involves the use of drugs that kill abnormally multiplying cells. The therapy uses one or more drugs in combination and…Anwesha Ghosh, PhD student in Biology, University of RochesterLicensed as Creative Commons – attribution, no derivatives.