An international study evaluated long-term outcomes from bone marrow-derived stem cell transplantation (autologous hematopoietic stem cell transplantation – HSCT) in 261 people with different forms of MS.

The transplants took place between 1995 and 2006, with a follow-up period of up to 16 years. Several different transplant protocols were followed.

Results suggest that after 5 years, 46% still had not experienced any progression or worsening of symptoms, including 73% of those with relapsing MS and 33% of those with secondary progressive MS. Eight deaths (2.8%) occurred within 100 days of the transplant. Most of these occurred during the early development of the procedure; improvements in patient selection and transplant techniques have significantly reduced the mortality.

Those with the best outcomes tended to be younger, had relapsing MS, lower accumulation of disability and had used fewer MS therapies prior to the transplant procedure.

Additional research is needed to better understand who might benefit from this procedure and how it compares to the benefits of powerful immune-modulating therapies now available.

The study, by Dr. Paulo Murano and colleagues, was published online February 20, 2017 in JAMA Neurology.

Background: HSCT (Hematopoietic Stem Cell Transplantation) attempts to “reboot” the immune system, which is involved in damaging the brain and spinal cord in MS. In HSCT for MS, hematopoietic (blood cell-producing) stem cells, which are derived from a person’s own (scientifically referred to as autologous”) bone marrow, are collected and stored, prior to depleting much of the immune system using chemotherapy drugs. Then the stored hematopoietic stem cells are reintroduced to the body. The new stem cells migrate to the bone marrow and over time reconstitute the immune system.

This Study: Researchers from 25 centers in 13 countries collaborated to evaluate the long-term outcomes from bone marrow stem cell transplantation (autologous hematopoietic stem cell transplantation – HSCT) in 261 people with different forms of MS. The researchers were part of the Multiple Sclerosis-Autologous Hematopoietic Stem Cell Transplantation Long-term Outcomes Study Group. The transplants took place between 1995 and 2006, with a follow-up period of up to 16 years. Several different transplant protocols were followed.

Characteristics of the participants varied widely in terms of age, amount of disability (as measured by the EDSS scale), and type of MS. Most had tried two or more MS therapies. More than 75% of the participants had progressive forms of MS at the time of the transplant, mostly secondary progressive MS but some with primary progressive MS.

Results: Results suggest that after 5 years, 46% still had not experienced any progression or worsening of symptoms, including 73% of those with relapsing MS and 33% of those with secondary progressive MS. Eight deaths (2.8%) occurred within 100 days of the transplant; most of these occurred in transplants made before 2000. Improvements in patient selection and transplant techniques seemed to have lowered the risk for mortality in subsequent years.

The investigators reported that those with better outcomes tended to be younger, had relapsing MS, less disability at the time of the transplant, and had used fewer MS disease-modifying therapies prior to the transplant procedure. By contrast, those who tended to experience MS progression after transplantation tended to be older, had progressive MS, and had tried more than two disease-modifying therapies prior to having the transplant.

Researcher Paolo A. Muraro, MD, PhD (Imperial College London) and colleagues published their results in JAMA Neurolology, published online on February 20, 2017.

This study was co-funded by many agencies including the UK Multiple Sclerosis Society.

“This is one of the larger studies to date that observed long-term outcomes of HSCT in MS,” says Bruce Bebo, PhD, the National MS Society’s Executive Vice President, Research. “Well-controlled trials are needed to better understand who might benefit from this procedure and how it compares to the benefits of the FDA-approved disease-modifying treatments that are now available.” A phase 3 trial of HSCT is now in planning stages. The Society is engaged with the team planning the trial and is encouraging quick action to design and launch the trial.