Stem cell transplants for people with MS: What does the research show?

Currently, stem cell transplants are an experimental treatment for MS. We know that they have the potential to help treat MS, but that the procedure can have a number of health risks. Research is ongoing to determine whether stem cell transplants are safe and effective.

Scientists first realized that stem cell transplants may work for MS when they discovered that MS patients, who underwent stem cell transplants as part of their cancer treatment, had a noticeable improvement in their MS. This, plus promising animal studies, led to more interest in studying stem cell transplants for people with MS.

Who's had a stem cell transplant?
Worldwide, more than 600 people with MS have had stem cell transplants as part of clinical studies. These studies have shown that the transplants may help delay disease progression, improve disability scores, reduce the number of relapses, decrease nerve cell inflammation, and reduce brain lesions. The hematopoietic stem cells also showed signs of moving into the brain and forming or protecting new nerve tissues. However, these benefits did not come without risks. The mortality (death) rate from stem cell transplants is approximately 1.3%. Many of these deaths were due to infection, bleeding, or disease progression. Another possible risk of transplant is graft versus host disease (GVHD). GVHD occurs when the transplanted cells attack the tissues of the recipient (the person receiving the transplant). It can lead to skin rashes, liver damage, and intestinal damage, and it may be fatal.

The Canadian MS/Bone Marrow Transplant Study Group is conducting a number of studies of stem cell transplants for people with MS. They have found that the transplants may decrease myelin destruction, which could help slow down the progression of MS.

Since most of the studies of stem cell transplants in MS have not been comparative (that is, they have not compared the transplant to another accepted treatment), comparative studies are now planned or ongoing. Also, many of the existing studies have been done in people with primary or secondary progressive MS rather than relapsing-remitting MS. Early studies in relapsing-remitting MS suggest treatment responses are better and longer in patients who received transplants during the relapsing-remitting phase than in those who received them in the secondary progressive phase.. In general, larger-scale comparative studies in people with all types of MS are needed before stem cell transplants can become a widely used treatment for MS.

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