U of M Medical School study team enrolls first Minnesotan in national clinical trial for stem cell transplant in multiple sclerosis
Study leaders will compare effectiveness of current FDA-approved medications against stem cell transplantation for severe, relapsing forms of the disease
MINNEAPOLIS/ST. PAUL (06/07/2021) — Physician-scientists from the University of Minnesota Medical School have enrolled Minnesota’s first patient in a nationwide clinical trial studying the best treatment option for severe forms of relapsing multiple sclerosis (MS). The disease impacts nearly a million people in the United States and is two to three times more likely to impact women, according to a 2019 study.
“They estimate that about 309 people every 100,000 in the United States have MS, but in the Midwest, that number is higher,” said the study’s lead investigator, Flavia Nelson, MD, a professor of neurology at the U of M Medical School and the director of the Multiple Sclerosis Division with M Health Fairview. “In the Midwest, we rank second in prevalence — the numbers increase to 353 people out of every 100,000 that are affected by this disease.”
M Health Fairview University of Minnesota Medical Center is one of 19 clinical trial sites — one of two in the five-state area — designated by the National Institute of Allergy and Infectious Diseases (NIAID) and the Immune Tolerance Network (ITN) to lead the research. All 19 are working together to study the safety, efficacy and cost-effectiveness of two therapeutic approaches.
“For the first time, we’re studying the differences between modern medications for MS approved by the Food and Drug Administration and an emerging treatment option using stem cell transplantation,” said study co-investigator, Claudio Brunstein, MD, PhD, a professor of medicine at the U of M Medical School and hematologist/oncologist and transplant physician with M Health Fairview. “There’s older evidence suggesting that stem cell transplantation is more effective, and people are traveling to other countries to get it. But, is it actually more effective than the current medications available, and if so, how? That’s what we’re studying.”
MS is an autoimmune disease where a person’s own immune cells attack the central nervous system. The experimental treatment under study — called autologous hematopoietic stem cell transplantation (or AHSCT) — uses a mixture of four chemical agents to remove these immune cells. Then, some of the person’s own blood-forming stem cells are infused back into their body. These stem cells repopulate the immune system, essentially resetting it so that the new immune cells no longer attack the central nervous system.
“Over the last few years, we’ve had highly effective medications for MS that were approved by the FDA. But, you have to take them indefinitely, and they are expensive,” Nelson said. “Not only are we studying whether or not one therapy is more effective than the other, but we’re also analyzing the cost of each. If the transplant costs a significant amount upfront, but it mitigates relapse or disease progression for several years, is it actually a more cost-effective treatment for the patient than if they paid several thousand dollars per year for these medications to do the same thing?”
For two years, Brunstein and Nelson had been preparing for this clinical trial, merging two scientific disciplines at the U of M Medical School in a novel way.
“The criteria to be a site for this trial was to have a very strong bone marrow transplant team and a very strong MS research team, so it was not easy for just any institution to qualify,” Brunstein said, who is also a member of the Masonic Cancer Center at the U of M. “To me, that’s a significant testament to the expertise of the Medical School and the M Health Fairview system and, now, a unique opportunity we can offer to our patients who have the severest form of this disease.”
BEAT-MS is sponsored by NIAID and conducted by the ITN under award number AI109565 and by the NIAID-funded statistical and clinical coordinating center under award number AI117870. The ClinicalTrials.gov identifier for the Phase 3 study Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) is NCT04047628.
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