Beat-MS Trial at University of Miami Tests Stem Cell Therapy for Treatment-Resistant Multiple Sclerosis
A national clinical trial led is evaluating whether autologous stem cell transplantation can improve outcomes for patients with relapsing-remitting multiple sclerosis that no longer responds to standard therapies.
A national phase 3 clinical trial now underway at the University of Miami Miller School of Medicine is evaluating whether an innovative, immune‑resetting approach may improve long‑term outcomes for people with treatment‑resistant relapsing‑remitting multiple sclerosis (RRMS).
Known as BEAT‑MS (ITN077AI), the multicenter, randomized controlled study compares autologous hematopoietic stem cell transplantation with best available therapy (BAT) in adults whose disease remains active despite prior use of high‑efficacy disease‑modifying therapies. The trial is designed to assess long‑term effectiveness, safety, immune system effects and cost‑effectiveness, with extended follow‑up.
At the Miller School, the study is led by neurologist Flavia Nelson, M.D., professor of clinical neurology at the Miller School and a nationally recognized expert in multiple sclerosis care and research. It is the only Beat-MS site in the state of Florida.
About the BEAT‑MS Trial
Multiple sclerosis is a chronic autoimmune disease in which the immune system attacks the central nervous system, damaging myelin and disrupting communication between the brain and body. While many people with MS benefit from disease‑modifying therapies, a subset continues to experience relapses and new MRI lesions, despite treatment.
The BEAT‑MS trial focuses specifically on this treatment-resistant population. Participants are randomly assigned to receive either autologous hematopoietic stem cell transplantation, an intensive procedure that aims to “reset” the immune system, or the best currently available MS therapies, selected by the treating neurologist (with the exclusion of the failed therapy).
“The BEAT‑MS study is designed to answer an important question for patients who continue to have active disease despite effective therapies,” said Dr. Nelson. “Can stem cell transplantation work where other treatments have not?”
To be eligible, participants must
• Be 18 to 55 years old
• Have a diagnosis of relapsing‑remitting multiple sclerosis based on 2017 McDonald Criteria
• Have an EDSS score of 6.0 or lower
• Have clinical and MRI evidence of ongoing disease activity
The study prioritizes individuals who have not responded adequately to disease modifying therapies based on experiencing at least two relapses within the past three years.
Understanding Autologous Stem Cell Transplant in MS
In autologous hematopoietic stem cell transplantation, a patient’s own stem cells are collected and later reintroduced after chemotherapy suppresses the immune system. The goal is to eliminate the immune cells driving MS activity and allow a healthier immune system to reestablish itself.
While autologous hematopoietic stem cell transplantation is not widely used as a first‑line MS treatment, it has drawn increasing interest for patients with highly active disease that does not respond to conventional therapies. BEAT‑MS is among the largest and most rigorous efforts to evaluate this approach against standard care.
“This trial allows us to carefully evaluate both effectiveness and safety of both autologous hematopoietic stem cell transplantation and high-efficacy MS treatments over the long term, which is exactly the kind of data patients and physicians need,” Dr. Nelson said.
A Community Investment in MS Research at the Miller School
As the BEAT‑MS trial advances research for people with aggressive multiple sclerosis, it does so amid growing national attention and philanthropic support for MS research at the University of Miami.
In April 2026, Miami native and Heisman Trophy winner Fernando Mendoza announced the launch of the Mendoza Family Fund, committing $500,000 to support multiple sclerosis research at UHealth and the Miller School, in partnership with the National Multiple Sclerosis Society. The initiative is deeply personal. Mendoza’s mother, Elsa Mendoza, has lived with MS for years.
The fund is designed to accelerate research efforts and support programs for individuals and families affected by MS, while also helping raise national awareness of the disease. The Mendoza family has committed to growing the fund to more than $3 million over the next three years.
By directing resources to UHealth and the Miller School, the Mendoza Family Fund reinforces the institution’s expanding role in advancing multiple sclerosis research, from clinical trials like BEAT‑MS to broader efforts focused on developing new treatment strategies.
Leadership in Multiple Sclerosis Research and Care
Dr. Nelson brings extensive clinical and research expertise to the study. Within the Miller School’s Multiple Sclerosis division, she has dedicated her career to improving diagnosis, treatment and outcomes for people with MS.
Her work emphasizes patient‑centered care and evidence‑based decision‑making, particularly for individuals with complex or refractory disease. Through her leadership on BEAT‑MS, Dr. Nelson and her team are reinforcing the Miller School’s position at the forefront of MS research.
“Our goal is always to offer patients clear, evidence‑based options and hope grounded in rigorous science,” she said.
More from the Miller School of Medicine
Two Miller School faculty were honored by SEQUINS for research in dementia and mentorship programs advancing neuroscience.
The Miami Project to Cure Paralysis wins the 2026 Health Care Heroes Award for advancing spinal cord research.
Miller School researchers at AD/PD 2026 highlighted why APOE is now a leading genetic target for Alzheimer’s treatment.
Ralph L. Sacco Research Day at the University of Miami spotlights resident and fellow neurology research and emerging clinician‑scientists.
Tags: Department of Neurology, Dr. Dipen Parekh, Dr. Flavia Nelson, genetics, immunology, John P. Hussman Institute for Human Genomics, MS, multiple sclerosis, neurology, philanthropy, stem cell therapies