New Relapsing MS Trial Seeks to Compare Stem Cell Transplant With Biologic Drugs


The trial will determine whether autologous hematopoietic stem cell transplantation is a more appropriate treatment option for patients with severe forms of relapsing MS compared to currently available biologic drugs.

Anthony S. Fauci, MD

Anthony S. Fauci, MD

The National Institutes of Health (NIH) has announced a new multiple sclerosis (MS) treatment trial comparing the safety, efficacy and cost-effectiveness of stem cell transplantation to the best available biologic drugs for severe forms of relapsing MS.

Although the FDA has approved over a dozen disease-modifying treatments for relapsing MS, some patients with severe forms of the disease fail to respond to first- and second-line therapies, leaving room for the development of more targeted, effective treatments.

“Autologous hematopoietic stem cell transplantation (AHSCT) has the potential to halt the progress of relapsing MS, eliminate the need for a person to take lifelong medication, and allow the body to partially regain function. However, we need to be certain that the benefits of this form of treatment outweigh its serious risks,” said Anthony S. Fauci, MD, director of the National Institute of Allergy and Infectious Diseases (NIAID), which is sponsoring the trial.

The trial, named BEAT-MS (BEst Available Therapy versus autologous hematopoietic stem cell transplant for Multiple Sclerosis [NCT04047628]) will be conducted by the NIAID-funded Immune Tolerance Network (ITN) and the Blood and Marrow Transplant Clinical Trials Network (BMT CTN).

The prospective, rater-masked, controlled trial will enroll 156 participants aged 18 to 55 across 19 sites in the United States and United Kingdom. Participants will be randomly assigned 1:1 to AHSCT or one of the high-efficacy biologic drugs. Investigators led by Jeffrey Cohen, MD, professor of neurology at the Cleveland Clinic Lerner College of Medicine, and the director of the Experimental Therapeutics Program in the Mellen Center for Multiple Sclerosis Treatment and Research at Cleveland Clinic, will assess levels of disability over the next 6 years.

The primary outcome is time from treatment assignment to MS relapse or death from any cause during the first 3 years of assessment. Additionally, investigators will note the development of the immune systems in patients under AHSCT compared to the patients who receive the biologic drugs. Lastly, the investigators will examine and compare measures such as disease activity and severity, quality of life, and cost effectiveness in terms of health care costs and individual productivity.

“We hope that BEAT-MS will clarify the best way to treat people with relapsing MS,” Cohen said in a statement.


New multiple sclerosis treatment trial compares stem cell transplantation to best available drugs [news release]. NIH. January 7, 2020. Accessed: January 7, 2020.

Related Videos
Patricia K. Coyle, MD
Video 2 - 5 KOLs are featured in "Natural History of Spinal Muscular Atrophy"
Video 1 - 5 KOLs are featured in "Clinical Features and Phenotypes of Spinal Muscular Atrophy"
Aliza Ben-Zacharia, PhD, DNP, ANP-BC, FAAN
 Brian G. Weinshenker, MD, FRCP
© 2024 MJH Life Sciences

All rights reserved.