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expert reaction to study looking at blood stem cell transplantation as a MS treatment

Research published in JAMA demonstrates that in patients with MS, Hematopoietic stem cell transplantation (HSCT),when compared with disease-modifying therapy (DMT), resulted in a longer time to diseases progression.

Dr Susan Kohlhaas, MS Society Director of Research, said:

“HSCT is an exciting development in MS treatment and we’ve seen incredible results for some people, but it’s also high-risk and won’t be right for everyone. We’re encouraged by the results of this study, but need to know how HSCT compares to the most effective disease modifying treatments for MS.

“As access to HSCT in the UK improves, our priority is making sure everyone who could benefit is able to access it. For some it has been life changing, and having that opportunity shouldn’t depend on your postcode.”

Professor Robin Franklin, Professor of Stem Cell Medicine, Wellcome – MRC Cambridge Stem Cell Institute, said:  

“These results look very promising and will strengthen the case for blood stem cell therapies in MS. However, it is important to emphasise that this is an approach where stem cells that form the blood target the immune system causing damage and is not a means of regenerating the damage that occurs in MS, which will require treatments using brain stem cells in some capacity.”

Professor Gavin Giovannoni, Professor of Neurology, Queen Mary University of London, said:

“This study doesn’t really tell us anything new regarding the promise of HSCT as a treatment for MS as it is an open-label study, where the researchers and participants know which treatment is being administered, which can bias the results.

“We also have concerns, as the stem cell transplants were compared to standard disease-modifying therapies that included some low and moderate efficacy treatments. Some high-efficacy treatments which are currently available were not included in this study, in particular alemtuzumab, which works in a similar way to stem cell transplantation.

“Overall the data supports haemopoetic stem cell transplantation being a very high-efficacy treatment, but it now needs to be compared to other high-efficacy treatments, including alemtuzumab, ocrelizumab and natalizumab, in a blinded randomised trial.”

‘Effect of nonmyeloablative hematopoietic stem cell transplantation vs continued disease-modifying therapy on disease progression in patients with relapsing-remitting multiple sclerosis: A randomized clinical trial’ by Bert et al. was published in JAMA at 16:00 UK time on Tuesday 15th January 2019.

All our previous output on this subject can be seen at this weblink:

Declared interests

None received. 

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