Stem Cell Transplant Trial Enrolls First Patient in Minnesota
The patient was enrolled at the University of Minnesota Medical School, one of two trial sites in the state. An additional 18 sites in the U.S. and one in the U.K. also are, or soon will be, recruiting participants for the BEAT-MS Phase 3 trial (NCT04047628). More information is available here.
The prevalence of MS is greater in Midwestern states than in the general U.S. population.
It is estimated that “about 309 people [of] every 100,000 in the United States have MS, but 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,” Flavia Nelson, MD, a professor of neurology and the study’s lead investigator, said in a press release.
More than a dozen disease-modifying therapies have been approved for the treatment of MS. However, in addition to their elevated costs, some of these therapies are not very effective at treating severe forms of relapsing MS.
A stem cell transplant has been explored as a treatment alternative for MS patients who failed to respond to conventional disease-modifying therapies (DMT). By reseting the immune system so that the central nervous system is no longer attacked, this would provide a more effective and longer-lasting alternative to patients.
However, whether a stem cell transplant — also known as an autologous (patient-derived) hematopoietic stem cell transplant — is more effective than conventional disease-modifying therapies remains unclear. Their safety and costs also have not been compared.
To address these questions, researchers designed BEAT-MS to compare the safety and effectiveness of a stem cell transplant to those of conventional therapies in patients with severe relapsing MS who failed two prior lines of therapy in the prior three years.
“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 Claudio Brunstein, MD, PhD, a professor of medicine at the University of Minnesota Medical School and a co-investigator in the trial.
“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,” he added.
BEAT-MS is enrolling up to 156 adults with mild-to-severe disability who will be assigned randomly to receive a stem cell transplant or high efficacy DMTs: Tysabri (natalizumab); alemtuzumab (sold as Campath and Lemtrada); Ocrevus (ocrelizumab); Mavenclad (cladribine); Kesimpta (ofatumumab); and rituximab (an experimental therapy used off-label in MS).
People assigned to the stem cell transplant will first receive medications that make stem cells leave the bone barrow and enter circulation, enabling their collection from the blood. Patients then are given chemotherapeutic agents that wipe out their entire immune system, after which the stem cells are infused back into the patient. These cells will originate all kinds of blood cells, reseting the immune system.
Patients will be followed for six years after their random assignments.
The trial’s main goal is to assess the length of time between a participant’s assignment to a treatment and MS relapse or death from any cause. Secondary goals include the number of relapses per year, evidence of MS activity, disability progression or improvement, mortality, and side effects.
Also, researchers will assess the cost-effectiveness of a stem cell transplant versus conventional DMTs.
“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?,” Nelson said.
BEAT-MS is sponsored by the National Institute of Allergy and Infectious Diseases (NIAID) and conducted by the Immune Tolerance Network (ITN).
Expertise in transplants of the bone marrow and in MS were two of the criteria to qualify as a clinical site for the trial, which precluded several institutions from joining.
“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,” said Brunstein.