What is stem cell therapy for MS?
When people talk about stem cell therapy, they usually mean autologous haematopoietic stem cell transplantation (abbreviated to AHSCT, HSCT or ASCT) which is more developed than other options being explored in clinical trials. It takes stem cells from your blood, freezes them and then reintroduces them into your body after your immune system has been partly, or completely, wiped out by chemotherapy. The idea is to rebuild your immune system so that it stops attacking your body and hopefully halts your MS symptoms. It is a relatively aggressive treatment based on a well-established blood cancer treatment. You can read more about stem cells, the theory behind stem cell treatments, how it is given and the results from clinical trials in MS so far.
The debate about stem cell therapy for MS
Stem cell therapy in MS is a topic that often generates debate and raises a lot of questions. For example, is it:
- A breakthrough in MS treatment?
- A cure?
- Still too risky by far?
- Suitable for everyone or just a select few?
Let’s look at these questions one by one.
Is it a breakthrough in MS treatment?
According to a review published in 2014, clinical trials have tested stem cell treatments in 538 people with MS. There are more clinical trials underway and some people have been treated outside of clinical trials. This all adds up to a relatively small number of people. The results so far are very encouraging but there is a long way to go.
So it still seems too early to say whether stem cell therapy is a breakthrough or not.
Is stem cell therapy a cure for MS?
According to the dictionary, a cure is “A drug or course of medical treatment used to restore health”. So, can stem cell treatment make MS go away? No, sadly not. In clinical trials, some people found that progression of their MS was halted for around two years after treatment as judged by their EDSS score, a common measure of disability. Longer term studies have suggested that progression of disability resumes for many people. This means that their MS has not been halted completely.
In clinical trials, only a small number of people experienced an improvement in their MS after treatment, as judged by their EDSS score. Undoubtedly, this improvement is great news for those individuals. However, it’s too early to know how much of this improvement will be maintained.
Consequently, stem cell treatment is not a cure for MS.
Is it too risky by far?
Well, that depends on your perspective. Like all treatments, it’s about balancing the risks and the benefits. Other treatments, including many of the disease modifying drugs, have longer established track records and are much safer. Stem cell therapy is in its infancy and there’s a risk of dying due to the procedure which currently runs at one or two people in every hundred (1.3%). That is much higher than any other MS treatment. Treatment protocols are improving all the time but there is still a lot of work to do to reduce the risks to a level that is acceptable to more people with MS and their health professionals.
Is it suitable for everyone or just a select few?
Some people with particularly aggressive MS are being referred for stem cell treatment. In these cases, the risks associated with treatment may be balanced out by the consequences of having such aggressive MS. Deciding where this balance lies is very much down to the person affected and their neurologist.
Whether stem cell treatment is appropriate for people with less aggressive MS is harder to say. Clinical trials show that only some people respond to stem cell treatment. Although it’s not clear cut, people are more likely to benefit if they have ongoing inflammatory disease (active lesions on MRI scans and/or continuing relapses despite treatment with disease modifying drugs) and do not have significant irreversible disability (usually 6.0 or less on the EDSS scale). However, there’s no guarantee that treatment will work so possible gains have to be weighed up against the known risks.
Current AHSCT treatment protocols are unlikely to work for people with progressive MS who have no ongoing inflammatory activity. Research has shown that the treatment will need adapting or, more likely, changing completely to tackle this aspect of the disease process. This is of huge importance and will be the subject of ongoing research.
What’s needed next?
The results from clinical trials are exciting as some people have experienced significant changes in their MS. However, stem cell therapy is a long way from being a routine treatment for MS. We need:
- To understand who is most likely to benefit from treatment so that others are not exposed to unnecessary risk for possibly little or no benefit
- To develop safer treatment procedures as the risk of death is still much too high compared with other treatment options
- To develop stem cell therapies that are more likely to work in people with progressive MS.
The way forward is to run more clinical trials as this is the only way to measure what works and what doesn’t.
Is stem cell therapy right for me?
Currently, stem cell therapy has not been formally assessed for use in the NHS outside clinical trials, although a few centres are able to provide AHSCT under specific circumstances to a very small number of people, generally after other treatments have failed.
If you, or someone you care about, are thinking about whether stem cell therapy may be a suitable treatment option, either now or in the future, it will be important to weigh up all the benefits and risks and to discuss all your options with your MS team, your family and friends. We have suggested some things to consider in our information about stem cell therapy. We hope you find them helpful.