The Immune Tolerance Network (ITN) has opened the BEAT-MS clinical trial for enrollment. BEAT-MS or “Best Available Therapy vs. Autologous Hematopoietic Stem Cell Transplant (AHSCT) for MS” will investigate high dose immunosuppression followed by AHSCT – a type of transplant using a patient’s own immune stem cells – compared to the best medical treatment currently available for multiple sclerosis (MS) in participants with relapsing MS.
MS affects nearly 1 million adults in the United States and an estimated 2.3 million individuals worldwide. MS is an autoimmune disease in which the body’s immune system attacks the protective myelin that covers nerve fibers in the brain and spinal cord and damages the nerve axons (fibers). As a result, nerve impulses are not transmitted as quickly or efficiently, resulting in symptoms such as difficulty walking, weakness, loss of vision and problems with memory, among other disabilities. Relapsing forms of MS are characterized by periods of time during which symptoms worsen (relapse), followed by periods of time in which symptoms get better (remission). Incomplete recovery from relapses often leads to worsening disability.
More than a dozen therapies have been approved by the FDA for the treatment of relapsing MS. However, despite this availability of an increasing number of treatment options, some patients with highly active disease do not achieve adequate disease control. In addition, side effects of therapy and the risk of relapse if therapy is stopped continue to be significant problems. Previous studies have shown that AHSCT could be an effective and durable treatment for patients with relapsing MS whose disease is incompletely controlled by the currently available therapies.
“Our earlier studies demonstrated the feasibility of AHSCT therapy in severe forms of relapsing MS, leading the way toward this important new trial that will evaluate durability of successful AHSCT treatment compared to alternative therapies,” noted Dr. Jerry Nepom, director of the ITN. “Replacing and restarting the immune system is potentially capable of stopping autoimmune diseases such as MS, but requires careful clinical trial evaluation to ensure the appropriate balance of risk and benefit for our patients.”
BEAT-MS will be the first clinical trial in which the efficacy, safety, and cost-effectiveness of AHSCT will be directly compared to the best available treatments. The best available treatments are chosen from among those recently approved for the treatment of MS that have not yet been tried by the study participant. Study participants assigned to the group receiving AHSCT will first have their own immune system stem cells mobilized out of the bone marrow, collected from the bloodstream, and then stored in a qualified laboratory for later use. Participants will then receive chemotherapy, which depletes the cells in their immune system that include those cells implicated in causing MS. After the chemotherapy, the participant’s own stem cells that were collected earlier will be given back to the participant to create a new population of immune cells.
“There is growing evidence that AHSCT can be a safe and effective treatment option for some people living with MS. The BEAT-MS study is expected to add to our knowledge about AHSCT by comparing the safety and effectiveness of this treatment directly with other powerful MS therapies in a well-designed and properly controlled study,” commented Bruce Bebo, PhD, Executive Vice President of Research for the National Multiple Sclerosis Society. “The results could change standards of care for people with some types of MS.”
BEAT-MS is being conducted by the ITN, a program of Benaroya Research Institute at Virginia Mason, funded by the National Institute of Allergy and Infectious Diseases (NIAID) under grant award number UM1AI109565, in collaboration with The Blood and Marrow Transplant Clinical Trials Network (BMT CTN). The first open clinical site that will begin recruiting participants for BEAT-MS is Cleveland Clinic. BEAT-MS will be conducted at 19 clinical sites across the United States and in the United Kingdom (U.K.). These sites include the Cleveland Clinic in Cleveland, OH, where Jeffrey Cohen, MD, serves as the protocol chair and lead investigator, in addition to the Fred Hutchinson Cancer Research Center in Seattle, WA, and Imperial College London in London, U.K., where the trial’s other two protocol co-chairs, George Georges, MD, and Paolo Muraro, MD, PhD, respectively, will lead the trial. The BMT CTN, funded by the National Heart Lung and Blood Institute and the National Cancer Institute, both components of NIH, is collaborating with the ITN to qualify participating transplant centers.