First Direct Comparison of Stem Cells vs Drugs in MS Underway

Damian McNamara

January 10, 2020

The first trial that will directly compare the safety and efficacy of stem cell transplantation to the best available drugs in the treatment of relapsing multiple sclerosis (MS) has launched.

Investigators are examining whether a one-time transplantation of autologous hematopoietic stem cells is superior to the best available biologic therapies, including natalizumab, alemtuzumab, ocrelizumab, and rituximab in the treatment of RRMS, the most common form of the disease.

"There is a lot of interest in transplant, but we think it's important to test it more comprehensively before it gains widespread use," principal investigator Jeffrey A. Cohen, MD, of the Mellen Center for Multiple Sclerosis at Cleveland Clinic in Ohio, told Medscape Medical News.

The US Food and Drug Administration (FDA) has approved more than a dozen drugs for the treatment of relapsing forms of MS. These agents vary in efficacy, safety, and cost. In addition, many first- and second-line medications fail to control the disease.

Previous studies suggest autologous hematopoietic stem cell transplantation [AHSCT] can have "very potent efficacy," Cohen said. "The efficacy is also durable — meaning after the transplant sometimes there is complete disease control for many years without any additional therapy."

However, AHSCT has never been formally tested in a head-to-head trial of available third-line drugs, which are effective but can have severe side effects. AHSCT also carries a risk of serious side effects, including death.

"With the transplant, almost all of the risk is at the time of the transplant, and then after the first 6 months, there is very little in terms of safety concerns. Whereas, with the medications, the risk accumulates while the person is on the medication," Cohen said.

Another potential advantage of AHSCT is cost-effectiveness over time, he said. "Transplant is quite expensive, roughly $150,000, but all of the cost is at the time of the transplant." The medications are also expensive, and the cost accumulates with use, Cohen added. "So, after about 2 to 3 years, transplant — if it turns out to be effective — is cost-effective."

Known as the Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) trial, the new study will enroll 156 MS patients ages 18 to 55 years from 19 sites in the United States and the United Kingdom.

The study's primary outcome measure is MS relapse-free survival, analyzed as time from treatment randomization until MS relapse or death from any cause up to 36 months.

Visit http://www.BEAT-MS.org for more information on the trial, including how to refer potential candidates.

Cohen has disclosed no relevant financial relationships.

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