SUMMARY: Investigators are recruiting 156 people with relapsing MS (relapsing-remitting MS or secondary progressive MS with relapses) at 19 sites nationwide for a pivotal clinical trial comparing autologous hematopoietic stem cell transplantation (AHSCT, also commonly known as HSCT) to disease-modifying therapies that have been shown to be the most highly effective in people with MS. This “BEAT-MS” study will be the first clinical trial in which the safety, efficacy, and cost-effectiveness of AHSCT will be directly compared to the best available treatments that have proven benefits for people with active relapsing MS.
“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 designed 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.”
DETAILS
Rationale: The goal of
AHSCT (Autologous Hematopoietic Stem Cell Transplantation) is to “reboot” the immune system, which is responsible for damaging the brain and spinal cord in MS. In AHSCT for MS, hematopoietic (blood cell-producing) stem cells, which are derived from a person’s own (scientifically referred to as “autologous”) bone marrow or blood, are collected and stored, prior to depleting much of the immune system using chemotherapy drugs. Then the stored hematopoietic stem cells are reintroduced to the body. The new stem cells migrate to the bone marrow and over time reconstitute the immune system. In many cases the ability of this new immune system to attack central nervous system myelin is greatly reduced or even eliminated along with MS relapses.
The BEAT-MS trial is being conducted by the Immune Tolerance Network and supported by the National Institute of Allergy and Infectious Diseases . The purpose of this well-controlled trial is to gather evidence needed to confirm AHSCT as a standard part of the roster of disease-modifying therapies available for people with MS. Unlike medications, this actual procedure does not need FDA approval. The medications used in AHSCT are commonly used chemotherapy medications. If positive, publication of the outcomes from this trial of AHSCT will encourage greater acceptance and use by the medical community and help pinpoint who would most benefit from this approach, compared to available disease-modifying therapies.
Eligibility and Details: Participants should be ages 18 to 55, diagnosed with MS, and experiencing highly active, treatment-resistant relapsing MS, defined as two or more episodes of treatment failure in the prior 36 months while taking medication for MS. Further enrollment criteria are available from the contacts at each study site.
All people enrolled in the trial will receive some form of treatment. Participants will be randomly assigned to undergo either AHSCT or to “best available therapy” – including either Tysabri
® (natalizumab, Biogen), Lemtrada
® (alemtuzumab, Genzyme, a Sanofi Company), Ocrevus
® (ocrelizumab - Genentech, a member of the Roche Group), Rituxan
® (rituximab - Genentech, a member of the Roche Group), Kesimpta® (ofatumumab, Novartis) or Mavenclad® (cladribine, EMD Serono). The primary outcome being measured is the length of time without relapses for up to 6 years (“relapse-free survival”) -. Secondary outcomes include changes in relapse rate, disability progression, the rate of brain tissue volume loss, levels of a molecule (neurofilament light chain) that reflects damage to the nervous system, and safety/adverse events.
Contact: To learn more about the enrollment criteria for this study, and to find out if you are eligible to participate, please contact the site nearest you, as listed on the study website,
www.beat-ms.org:
Sites are or will soon be recruiting in the following cities:
Palo Alto, California
Aurora, Colorado
Worcester, Massachusetts
Minneapolis, Minnesota
Rochester, Minnesota
Saint Louis, Missouri
Buffalo, New York
New York, New York
Rochester, New York
Durham, North Carolina
Cincinnati, Ohio
Cleveland, Ohio
Portland, Oregon
Philadelphia, Pennsylvania
Dallas, Texas
Houston, Texas
Richmond, Virginia
Seattle, Washington
Download a brochure that discusses issues to think about when considering enrolling in an MS clinical trial (PDF).
Without participants in research studies, MS research would come to a standstill.
Read more here.
Read more about AHSCT in MS
Tysabri is a registered trademark of Biogen
Lemtrada is a registered trademark of Genzyme, a Sanofi Company
Ocrevus is a registered trademark of Genentech, a member of the Roche Group
Rituxan is a registered trademark of Genentech, a member of the Roche Group
Kesimpta is a registered trademark of Novartis
Mavenclad is a registered trademark of EMD Serono