Target Health Blog

Stem Cell Transplants May Induce Long-Term Remission of Multiple Sclerosis

February 6, 2017

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Neurology
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Multiple sclerosis (MS), is an autoimmune disease in which the immune system attacks the central nervous system. MS symptoms vary widely and may include motor and speech difficulties, weakness, fatigue and chronic pain. The most common form of MS is relapsing-remitting MS, which is characterized by periods of mild or no symptoms interspersed with symptom flare-ups or relapses. Over years, the disease can worsen and shift to a progressive form.

According to an article, published online (1 February 2017) in Neurology, the medical journal of the American Academy of Neurology, new clinical trial results provide evidence that high-dose immunosuppressive therapy followed by transplantation of a person’s own blood-forming stem cells can induce sustained remission of relapsing-remitting MS. Study results showed that 5 years after receiving the treatment, called high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT), 69% of trial participants had survived without experiencing progression of disability, relapse of MS symptoms or new brain lesions. Notably, participants did not take any MS medications after receiving HDIT/HCT. Other studies have indicated that currently available MS drugs have lower success rates.

The trial, called HALT-MS, was sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and conducted by the NIAID-funded Immune Tolerance Network (ITN). The researchers published three-year results from the study in December 2014, and the final five-year results were published this month.

The HALT-MS study tested the safety, efficacy and durability of HDIT/HCT in 24 volunteers aged 26 to 52 years with relapsing-remitting MS who, despite taking clinically available medications, experienced active inflammation, evidenced by frequent severe relapses, and worsened neurological disability. The experimental treatment aimed to suppress active disease and prevent further disability by removing disease-causing cells and resetting the immune system. During the procedure, doctors collected a participant’s blood-forming stem cells, give the participant high-dose chemotherapy to deplete the immune system, and return the participant’s own stem cells to rebuild the immune system. The treatment carries some risks, and many participants experienced the expected side effects of HDIT/HCT, such as infections. Three participants died during the study; none of the deaths were related to the study treatment. Five years after HDIT/HCT, most trial participants remained in remission, and their MS had stabilized. In addition, some participants showed improvements, such as recovery of mobility or other physical capabilities.

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