A stem cell trial is testing whether transplantation can improve outcomes for people with treatment-resistant relapsing MS.
The phase 3 study is comparing autologous haematopoietic stem cell transplantation with the best available therapy for adults whose disease remains active despite previous high-efficacy disease-modifying treatments.
Relapsing-remitting multiple sclerosis, or RRMS, is a form of MS where symptoms flare up and then partly or fully improve.
MS is a long-term autoimmune condition in which the immune system attacks the brain and spinal cord, damaging the protective coating around nerves and disrupting signals between the brain and body.
The BEAT-MS trial is a multicentre, randomised controlled study, meaning participants are assigned to different treatment groups so researchers can compare outcomes.
The study is designed to assess long-term effectiveness, safety, immune system effects and cost-effectiveness, with extended follow-up.
At the University of Miami Miller School of Medicine, the trial is being led by Dr Flavia Nelson, professor of clinical neurology and a specialist in MS care and research.
It is the only BEAT-MS site in Florida.
The study is focused on adults with relapsing-remitting MS who continue to have relapses or new MRI lesions despite high-efficacy disease-modifying therapies.
MRI scans use magnetic fields to create detailed images of the brain and spinal cord, helping clinicians see signs of disease activity.
Participants are randomly assigned to receive either autologous haematopoietic stem cell transplantation or the best available MS treatment, selected by their neurologist. The failed therapy is excluded from the best available therapy option.
Autologous haematopoietic stem cell transplantation uses a patient’s own stem cells.
The cells are collected and later returned after chemotherapy suppresses the immune system, with the aim of reducing the immune activity driving MS.
Dr Nelson said: “The BEAT-MS study is designed to answer an important question for patients who continue to have active disease despite effective therapies. Can stem cell transplantation work where other treatments have not?”
To be eligible, participants must be aged 18 to 55, have a diagnosis of relapsing-remitting MS based on 2017 McDonald Criteria, have an EDSS score of 6.0 or lower and show clinical and MRI evidence of ongoing disease activity.
The EDSS, or Expanded Disability Status Scale, is a standard measure used to assess disability in people with MS.
The study prioritises people who have not responded adequately to disease-modifying therapies, including those who have experienced at least two relapses in the past three years.
While autologous haematopoietic stem cell transplantation is not widely used as a first-line MS treatment, it has attracted increasing interest for people with highly active disease that does not respond to conventional therapies.
Dr Nelson said: “This trial allows us to carefully evaluate both effectiveness and safety of both autologous haematopoietic stem cell transplantation and high-efficacy MS treatments over the long term, which is exactly the kind of data patients and physicians need.”
The trial is taking place amid wider investment in MS research at the University of Miami.
In April 2026, Miami native and Heisman Trophy winner Fernando Mendoza announced the Mendoza Family Fund, committing US$500,000 to support MS research at UHealth and the Miller School, in partnership with the National Multiple Sclerosis Society.
Mendoza’s mother, Elsa Mendoza, has lived with MS for years.
The fund is intended to accelerate research and support programmes for people and families affected by MS, while raising awareness of the condition.
The Mendoza family has committed to growing the fund to more than US$3m over the next three years.
Dr Nelson’s work within the Miller School’s MS division focuses on diagnosis, treatment and outcomes for people with the condition, particularly those with complex or treatment-resistant disease.
Dr Nelson said: “Our goal is always to offer patients clear, evidence-based options and hope grounded in rigorous science.”
