Multiple sclerosis (MS) sufferer Eric Thompson was able to rise from his wheelchair and walk just days after treatment with haematopoietic stem cells, according to a recent Daily Mail article. Unable to receive this aggressive treatment through the NHS, Thompson and his family raised £40,000 to undergo the treatment in Mexico.
The treatment Thompson received is called autologous haematopoietic stem cell transplantation (AHSCT). It involves extracting stem cells from the affected person’s bone marrow or blood. After the person has undergone intensive chemotherapy to wipe out their immune system, the stem cells are then reintroduced. It’s done to try to reboot the immune system. The use of intensive high-dose chemotherapy and the transplant process is an arduous procedure for this potent treatment.
According to Cancer Research UK donor stem cell transplants are used as a treatment for some types of cancer, including leukaemia, lymphoma and myeloma. However, it’s probably premature to recommend AHSCT as a treatment for MS.
Although it is not available on the NHS, the MS Society UK report a number of studies using the treatment for MS and they have shown promise, demonstrating a reduction in relapse for some people, stabilisation and even some reversal of disability. However, the MS Society also highlights that this is not the case for every MS patient. The treatment is aggressive and has significant risks.
A number of AHSCT trials for MS are taking place, including the ASTIMS trial, published March 2015. This was conducted on 21 patients with relapsing remitting MS (individuals have distinct bouts of symptoms) or secondary progressive MS (disability builds up independent of relapses). Those having AHSCT showed a significantly greater reduction in relapses compared to patients having mitoxantrone.
Mitoxantrone is a type of chemotherapy treatment sometimes used as a disease-modifying therapy to reduce the number of relapses a person with MS might have. Researchers did, however, also state that some patients having AHSCT experienced severe adverse effects, such as thrombocytopenia (low platelet levels), neutropenia (low white blood cells), infection, and liver toxicity, with two of the participants suffering from life-threatening conditions including sepsis, severe infections and either a slow take-up of stem cells by the bone marrow (“engraftment”) or a complete failure to do so.
The UK is part of the international multi-centre (UK, US, Sweden and Brazil) randomised clinical trial MIST and several patients having AHSCT have shown particularly encouraging results. They reported improvement and reversal of MS symptoms, which were backed up by MRI results demonstrating no evidence of active disease. The full results of the trial are due to be published in 2017 and a lot of people are waiting with interest. The UK centre is the Royal Hallamshire Hospital in Sheffield and some of their trial patients were featured in a Panorama programme in January 2016.
A clinical trial being undertaken in Canada using AHSCT has also demonstrated clear changes in relapse rate and disease activity, with 70% of participants showing a complete stop in disease progression. But those running the trial also reported the death of one patient due to liver failure, a side effect of the treatment. They highlight that because of the associated risks this treatment should only be given to those with active aggressive MS.
Thompson’s story highlights real promise in the potential of AHSCT treatment, but it does not flag up the risks involved and suggests the only drawback is the cost. It is important that people take note of Sorrel Bickley, head of biomedical research at the MS Society, who commented: “This is an aggressive procedure that comes with substantial risks, requires specialist aftercare and should be carried out at an accredited centre.” The long-term effects of AHSCT treatment are not known, further highlighting the importance of clinical trials.