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Multiple Sclerosis Centers of Excellence

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Stem Cell Therapy for Multiple Sclerosis

Derek McFaul, DO and Rebecca Spain, MD, MSPH

MS is an autoimmune disease that attacks myelin, the insulating cover of cells in the brain and spinal cord. Stem cell therapy is an experimental procedure being studied in MS to “reset” the immune system so that it is less likely to attack myelin. Since the first human studies in 1995, stem cell therapy has shown great potential for preventing MS disease activity, but with risks of serious complications. Ongoing studies are trying to determine which people with MS will benefit most from stem cell therapy, and which stem cell therapy methods are safest and most effective.

Studies of stem cell therapy for MS generally use a type of stem cell therapy called autologous hematopoetic stem cell therapy (aHSCT). Autologous means the stem cells used for treatment are taken from the treated person’s own body. Hematopoetic stem cells (HSC) are adult stem cells found in bone marrow and blood, and are capable of producing all of the cells that make up the blood and immune system. The process of aHSCT starts with medications that release stem cells from the bone marrow into the blood. These stem cells are then collected and frozen. This is followed by a treatment course of high-dose chemotherapy to eradicate the remaining immune system. Finally, the previously collected stem cells are infused back into the person’s blood and stimulated to repopulate the person’s immune system “from scratch.”

This one-time process usually takes place in the hospital over several weeks. You can read more details of the procedure at this National MS Society webpage including what happens during the treatment, after treatment, risks and benefits of stem cell therapy, finding stem cell treatments, and resources to learn more. 

Still being investigated is how and when to use aHSCT to treat MS and the safety of aHSCT compared to other MS therapies. The BEAT-MS study, currently ongoing at multiple sites across the United States, will help answer this question in people with aggressive MS who are not responding optimally to available MS medications. Currently, aHSCT remains experimental for MS and the VA does not offer stem cell therapy for people with MS. The VA MS Centers of Excellence strongly urge Veterans with MS to avoid getting aHSCT from for-profit and unaccredited stem cell centers. We encourage those interested in considering aHSCT to discuss it further with their physician and consult the resources below. 

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