Experimental Treatment Induces Remission in Multiple Sclerosis

A new treatment option using stem cell transplants was shown to induce sustained remission of relapsing-remitting multiple sclerosis (MS). More than two-thirds (69%) of patients in the HALT-MS trial who received high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT) had no signs of progression of disability, relapse of MS symptoms, or new brain lesions after 5 years.

The trial was sponsored by the National Institutes of Health (NIH)’s National Institute of Allergy and Infectious Diseases (NIAID) and conducted by the Immune Tolerance Network. Initial results from the trial were published in December 2014, but the 5-year results were published February 1, 2017, in Neurology.

“These extended findings suggest that one-time treatment with HDIT/HCT may be substantially more effective than long-term treatment with the best available medications for people with a certain type of MS,” NIAID Director Anthony S. Fauci, MD, said in a statement. “These encouraging results support the development of a large, randomized trial to directly compare HDIT/HCT to standard of care for this often-debilitating disease.”

A total of 24 participants—median age 37 years and 68% women—underwent HDIT/HCT. The participants had previously failed 3 nontransplant MS medications. Two participants had disease progression and died after the transplant, while a third participant died 4.5 years after transplant. The deaths were not attributed to the treatment, though.

NIH reported that after the 5 years, most participants remained in remission and that their MS had stabilized. In addition, participants showed improvements in neurological disability.

“If these findings are confirmed in larger studies, HDIT/HCT may become a potential therapeutic option for patients with active relapsing-remitting MS, particularly those who do not respond to existing therapies,” said Daniel Rotrosen, MD, director of NIAID’s Division of Allergy, Immunology and Transplantation.

The purpose of HDIT/HCT is to suppress active disease and prevent further disability. During the procedure, doctors remove disease-causing cells, deplete the patient’s immune system with high-dose chemotherapy, and then return the collected stem cells.

“For patients failing first-line treatments, significantly more potent options are becoming available,” the authors concluded in the study. “We suggest that HDIT/HCT may be a reasonable consideration for such patients.”