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Stem Cell Transplants May Halt Progression of Multiple Sclerosis

NIH-funded study yields encouraging early results

Three-year outcomes from an ongoing clinical trial suggest that high-dose immunosuppressive therapy followed by the transplantation of a person’s own blood-forming stem cells may induce sustained remission in some people with relapsing-remitting multiple sclerosis (RRMS). RRMS is the most common form of MS, a progressive autoimmune disease in which the immune system attacks the brain and spinal cord.

The trial was funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.

Three years after the treatment, called high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT), nearly 80% of trial participants had survived without experiencing increased disability, a relapse of MS symptoms, or new brain lesions. The investigators observed few serious early complications or unexpected adverse effects, although many participants experienced expected side effects of high-dose immunosuppression, including infections and gastrointestinal problems.

The findings were published online December 29 in JAMA Neurology.

“These promising results support the need for future studies to further evaluate the benefits and risks of HDIT/HCT and to directly compare this treatment strategy to current MS therapies,” said NIAID Director Anthony S. Fauci, MD. “If the findings from this study are confirmed, HDIT/HCT may become a potential therapeutic option for people with this often-debilitating disease, particularly those who have not been helped by standard treatments.”

Scientists estimate that MS affects more than 2.3 million people worldwide. Symptoms of the disorder can vary widely and may include disturbances in speech, vision, and movement. Most people with MS are diagnosed with RRMS, which is characterized by periods of relapse or flare-up of symptoms followed by periods of recovery or remission. Over years, the disease can worsen and shift to a more progressive form.

In the new study, researchers tested the effectiveness of HDIT/HCT in 25 volunteers with RRMS who had relapsed and had experienced worsened neurologic disability while taking standard medications. Physicians collected blood-forming stem cells from participants and then gave them high-dose chemotherapy to destroy their immune systems. The doctors then returned the stem cells to the participants to rebuild and reset their immune systems.

“Notably, participants did not receive any MS drugs after transplant, and yet most remained in remission after 3 years,” said Daniel Rotrosen, MD, director of the NIAID’s Division of Allergy, Immunology, and Transplantation. “In contrast, other studies have shown that the best alternative MS treatments induce much shorter remissions and require long-term use of immunosuppressive drugs that can cause serious side effects.”

The researchers plan to follow the participants for a total of 5 years, recording all adverse events associated with the treatment. Final results from this and similar studies promise to help inform the design of larger trials to further evaluate HDIT/HCT in people with MS, the researchers say.

Source: NIH; December 29, 2014.

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