A recent study (JAMA Neurol. 2017; 74(4):459-469) by Paolo Muraro, et al. looks at long-term results of stem cell therapy on multiple sclerosis (MS). This study followed 281 MS patients with predominantly progressive forms of the disease that were not responding to standard therapies. These patients received autologous hematopoietic stem cell transplants between 1995 and 2006 (meaning that the stem cells were taken from their own peripheral blood).
The observational data was collected from 25 different medical facilities in 13 countries, with 77% of the patients exhibiting progressive forms of MS and median follow-up was 6.6 years. Multiple factors were looked at in this study, including age, number of prior disease-modifying treatments, disease sub-type/severity, baseline Expanded Disability Status Scale (EDSS) score, and the overall intensity of conditioning regimen and graft manipulation. The primary outcomes were both MS progression-free survival and overall survival.
Out of the 281 patients, 8 died within the first 100 days of transplant and these were considered a transplant-related mortality of 2.8%. It is not known if these deaths were truly attributable to the stem cell therapy, but were considered for mortality based on the structure of the study.
The 5-year probability of progression-free survival was 48% and overall 5-year survival was 93%. It is remarkable that almost half of the patients remained free from neurological progression at 5 years. Better outcomes were associated with younger age (not surprising, as this has been repeatedly shown), relapsing form (vs. progressive MS), fewer previous immunotherapies, and lower baseline EDSS scores.
These findings support further studies of stem cell therapies for treatment of MS in order to delineate the exact modalities and procedures that will provide consistent and beneficial results. Once again, stem cells seem to be a key link in the future of medicine and treatment of difficult disease processes.
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