Landmark trial finds stem cell treatment reduces disability for those with multiple sclerosis
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Pioneering new research assessing the effectiveness of stem cell treatment for multiple sclerosis (MS) has found that the procedure can stabilize the disease and improve disability.
Led by Northwestern University in Chicago, the research is the first ever international large scale randomized trial looking at the effect of autologous hematopoietic stem cell transplantation (AHSCT) in relapsing remitting multiple sclerosis (MS).
Article continues after this advertisementAHSCT is an intensive treatment which resets a patient’s immune system to a point before it caused MS, using stem cells harvested from their own blood and bone marrow. It is currently only suitable for patients with the relapsing remitting form of the disease who haven’t responded to standard treatments and who have lived with the disease for ten years or less.
For the new trial the researchers recruited 110 MS patients who met the criteria and randomly assigned half of them to receive AHSCT, while the other half received the best available drug treatment.
The disability of each participant was measured using a standard assessment tool known as the Expanded Disability Status Scale (EDSS).
Article continues after this advertisementA score of 1.0 to 4.0 indicates that an individual is still able to walk but has some difficulties with vision, movement, sensation, coordination and bladder control. A score of 4.5 to 6.5 indicates difficulties with mobility, while those at the higher end of the scale have more severe disability and may be confined to their bed.
During the follow-up period, those who had received AHSCT showed a significant improvement in their disability level, with the EDSS score of these patients improving from an average of 3.5 to 2.4, which is unprecedented in MS treatment trials.
However, those who received the drug treatment actually saw their EDSS scores decline, from an average score of 3.3 to 3.9.
In addition, after 30 patients who started in the drug treatment group were moved over to the AHSCT group, following a decline in their EDSS scores, the team also observed that their disability scores improved, from 5.2 to 2.6.
The researchers also found that within a year of joining the trial only one patient in AHSCT group suffered a relapse, compared to 39 relapses in the group receiving the drug treatment, and no patients in the AHSCT arm suffered any significant side effects.
Co-investigator Professor Basil Sharrack from Sheffield’s Royal Hallamshire Hospital commented on the research saying, “We are very excited by these hugely encouraging findings, which are the first to assess the long-term effectiveness of AHSCT in people with the active relapsing remitting form of the disease in a phase three randomised trial.”
Professor John Snowden, also at the University of Sheffiled, added, “The initial results of the MIST trial show that this type of stem cell transplant can be delivered with acceptable safety to people with highly active relapsing remitting MS.”
“However, longer-term evaluation is necessary and patients treated in the MIST trial will be followed up until 2021,” said Snowden. “It is important to stress that this treatment is unfortunately not suitable for every person with MS. This type of stem cell transplant targets the inflammatory phase of MS.” JB
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