Autologous hematopoietic stem cell transplantation (HSCT) has sparked global interest in recent months. In MS, HSCT works by “resetting” the diseased immune system and replacing it with a healthy one.

A number of studies have shown that the treatment has the potential to stop the progression of MS in its tracks, prevent the requirement of disease modifying therapies (DMTs), and in some cases improve function. However, only limited information has been available about how people do in the longer term following a transplant.

Researchers around the world are continuing to study HSCT as a potential treatment for MS, and a new study looks further into these trials and what they can tell us about the long term benefits and risks of HSCT.

The Study

The study looked at a group of people living with MS who received HSCT between 1996 and 2006. Researchers analysed data from 281 participants from 25 centres across 13 countries that underwent the treatment. They examined demographic and clinical data (EDSS score, type of MS, treatment history, as well as the type of chemotherapy regimen that was administered- high, intermediate, or low intensity). The researchers looked at the proportion of people who had progression-free survival, meaning their disability level stayed the same before and after the treatment. They based this assessment on the observed changes in Expanded Disability Status Score (EDSS).


MS progression was halted in 46% of all individuals at 5 years post-HSCT. The study also determined that the progression of MS after the HSCT is more likely to occur in (1) older versus younger people with MS (2) those with progressive MS versus relapsing-remitting MS and (3) individuals that previously were on more than two DMTs. For example, 73% of people with relapsing-remitting MS compared with 33% of people with secondary progressive MS experienced an absence of disease progression at the 5 year post-treatment assessment.

In a subgroup of 111 people that were further analysed, those individuals with relapsing-remitting MS showed an improvement in disability following HSCT (EDSS change of -0.76), and for people with progressive MS improvement was also observed but to a lesser degree (EDSS change of -0.14).

There were 8 deaths reported within the first 100 days after transplant, which were related to the treatment.

The findings from this study provide insight into the long-term impact of HSCT in people living with all forms of MS, and serves as a driving force for larger, well-designed trials that could compare HSCT with other available DMTs or against a placebo treatment. It provides information that will help guide decisions of people with MS and health care professionals who are considering HSCT, and paves the way for ongoing research in the application of stem cells for MS.

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