BEAT-MS: new trial to compare efficacy and costs of stem cell treatment for MS versus gold-standard biologics
A new study, funded by the National Institutes of Health (MD, USA), will investigate the comparative effectiveness of an experimental hematopoietic stem cell treatment versus the current gold-standard biologic therapies for certain individuals with severe forms of relapsing multiple sclerosis.
Researchers from the National Institute of Allergy and Infectious Diseases, a division of the National Institutes of Health (MD, USA), have launched the BEst Available Therapy versus autologous hematopoietic stem cell transplant for Multiple Sclerosis (BEAT-MS) trial. This new study will investigate the comparative safety, efficacy and cost–effectiveness of an experimental stem cell treatment versus current gold-standard biologic therapies for individuals with severe forms of relapsing multiple sclerosis (MS) who have failed to respond to first- or second-line treatments.
MS is an autoimmune disease – affecting approximately 2.3 million individuals worldwide – in which individuals’ immune systems attack myelin-producing cells in the central nervous system; myelin is essential for salutatory conduction along nerve axons and rapid neuronal communication.
The experimental, autologous hematopoietic stem cell treatment for MS to be evaluated in this trial comprises the targeted removal of immune cells responsible for myelin loss with a cocktail of four chemical mediators. Autologous hematopoietic stem cells are then injected into individuals to repopulate the cells of the immune system with ones not primed for myelin degradation.
The US FDA has already approved various biologic agents for the management of relapsing MS, which vary in their associated costs and efficacies. While previous research has suggested that autologous hematopoietic stem cell transplantation could be an effective treatment for individuals who fail to respond to first-line treatments for relapsing MS, the direct comparative effectiveness of the approach versus current gold-standard, third-line treatments has not be evaluated.
BEAT-MS is to enroll a total of 156 study participants, aged 18–55 years, across 19 sites in the UK and USA. Participants will be randomized to receive either the experimental, autologous hematopoietic stem cell treatment or a best-available, third-line biologic drug. Study participants will be followed for 6 years and will be regularly assessed by neurologists, who will not know the treatment the individuals are receiving.
The primary study outcome will be time to MS relapse or all-cause mortality during the first 3 years of follow-up. Additional treatment effects, such as those on other measures of disease activity and severity, cost–effectiveness and participants’ quality of life will also be evaluated.
Anthony Fauci, Director of the National Institute of Allergy and Infectious Diseases, explained: “[Autologous hematopoietic stem cell transplantation] 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.”
Trial lead Jeffrey Cohen (Cleveland Clinic Lerner College of Medicine; OH, USA) stated: “We hope that BEAT-MS will clarify the best way to treat people with relapsing MS.”