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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 as a way 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 which are capable of producing all of the cells that make up the blood and immune system. The process of autologous hematopoetic stem cell therapy 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 chemotherapy to suppress the remaining immune system. Finally, the previously collected stem cells are infused back into the patient’s blood.

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 (NMSS) webpage. A 2017 analysis of 15 individual studies on aHSCT in MS found that 5 years after the procedure, between 59-70% of people had no evidence of MS disease activity, while the combined mortality (death) rate from the procedure was 2.3%. Further analysis of this data showed that those with relapsing remitting MS had better outcomes and safety compared to those with progressive MS. Importantly, the newer trials had lower rates of complications, further evidence that more time and experience with these procedures increases the safety.

Currently, stem cell therapies for MS are available in the context of clinical trials and at accredited stem cell centers. In recent years there has also been an increasing number of for-profit and non-accredited stem cell treatment centers offering this procedure as well increasing “stem-cell tourism” where people travel to other countries to receive stem cell therapy. A study in 2016 raised concerns about the lack of standardized safety protocols at many of these sites, emphasized the need for better oversight, and raised ethical and regulatory concerns related to marketing of unproven treatments. The NMSS has compiled a list of important questions to ask when researching a stem cell therapy center for treatment, including asking about the facility accreditation, medical team experience and complication rates, and knowing their plan for follow up after the procedure.

Still unknown is how effective aHSCT is at treating MS compared to some of the most effective MS therapies like natalizumab (Tysabri), ocrelizumab (Ocrevus), and alemtuzumab (Lemtrada). The BEAT-MS study, currently ongoing at multiple sites across the United States, will help answer this question as well as further study the safety of aHSCT in people with aggressive MS who are not responding optimally to available MS medications. At this time, the VA MS Centers of Excellence strongly urge Veterans with MS to avoid getting aHSCT from for-profit and unaccredited stem cell centers. Instead, we encourage those interested in considering this treatment to discuss it further with their physician, including joining clinical trials, while we wait for further evidence for how and when to best utilize aHSCT to treat MS.

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