Reasons for hope
The trial involved 24 people whose immune systems were completely wiped out with chemotherapy and then rebooted with their own stem cells - an approach called autologous haematopoietic stem cell therapy (AHSCT). Most people in the trial did incredibly well in all the measures that the study focussed on. Their mobility didn’t worsen, they had no signs that their disease was active on brain scans and they had no new relapses. This was over a long period as well – for some participants there is 12 years of evidence.
The participants, as in any trial, were selected according to a clear set of criteria. They had either relapsing remitting MS or were early into secondary progressive MS. In either case, they still had active inflammation. They were also still walking, at most using one stick. Nonetheless, most of the participants benefitted hugely from the treatment and this is something we should welcome and indeed celebrate. It’s a very positive result.
A heavy price
We have to recognise that one participant in this small trial died as a direct result of the aggressive chemotherapy given as part of the stem cell therapy regime. Our hearts go out to the family of this individual. It is hugely tragic and just drives home the scale of the risk that is still inherent in this treatment. No person with MS, no neurologist and no haematologist undertakes a powerful and still experimental treatment lightly. These are major and very personal decisions and anyone thinking about stem cell therapy needs to stick to trusted sources of information when considering their options and only consider well-established treatment centres that are safe, well-monitored and can offer proper follow-up after treatment, when many serious problems can arise.
The death of the participant was within the context of a trial, which means that even with maximum amounts of safety checks and close monitoring, the scale of the risk could not be eliminated. In less controlled environments, the risks of devastating outcomes are even greater, so we do urge huge caution for anyone who is considering whether stem cell therapy might be right for them.
Where do we go from here?
The MS Trust believes that stem cell therapy holds hope for the future. That’s why we have contributed funding to the ACTIMUS trial, looking at one form of stem cell therapy (different from the one in this trial) and, more importantly why we are funding the first UK audit of the pan-London stem cell service. We want to support real world data about how stem cell services can be developed in the UK, including how to select the participants who will most benefit from the treatment, what a good service should provide and follow-up data on how they do over time.
We believe the time has come for the MS community to focus their efforts on laying out the roadmap to HSCT becoming more widely available on the NHS. We need to
- identify which type of stem cell therapy most effectively balances risks and benefits
- define the groups of people with MS who would gain greatest benefit from the treatment, particularly in light of the risks
- identify the safety precautions which need to be in place to minimise the risks as much as possible
- work with researchers, those delivering MS specialist services and NHS bodies to chart the route to establishing stem cell therapy services in the UK
- develop information resources for people with MS so they can understand their options and make the choices that are right for them
The MS community has faced this kind of challenge before. There have been other disease modifying therapies that have had a potent mix of benefit and risk (including death) and we have, together, found a way forward that made those treatments available with the maximum amount of safety possible. The MS Trust is committed to finding a way to do it again for this treatment, which could bring real hope to many people with MS.