High-dose immunosuppressive therapy followed by transplantation of a person’s own blood-forming stem cells can induce sustained remission of relapsing-remitting multiple sclerosis (MS), an autoimmune disease in which the immune system attacks the central nervous system, new clinical trial results show.
Five years after receiving the treatment, called high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT), 69 percent 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.
Hematopoietic Cell Transplant
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).
“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,” said NIAID Director Anthony S. Fauci, M.D. “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.”
Multiple sclerosis 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.
Five Years Remission
In the HALT-MS trial, researchers tested the safety, efficacy and durability of high-dose immunosuppressive therapy and autologous hematopoietic cell transplant 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 aims to suppress active disease and prevent further disability by removing disease-causing cells and resetting the immune system. During the procedure, doctors collect 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.
“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, M.D., director of NIAID’s Division of Allergy, Immunology and Transplantation.
Richard A. Nash, George J. Hutton, Michael K. Racke, Uday Popat, Steven M. Devine, Kaitlyn C. Steinmiller, Linda M. Griffith, Paolo A. Muraro, Harry Openshaw, Peter H. Sayre, Olaf Stuve, Douglas L. Arnold, Mark H. Wener, George E. Georges, Annette Wundes, George H. Kraft, James D. Bowen High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS Neurology, 2017; 10.1212/WNL.0000000000003660 DOI: 10.1212/WNL.0000000000003660