Immune System Reboot More Effective for MS

By Christina Phillis

Multiple sclerosis (MS) is a complicated disease. Outwardly, patients exhibit vision loss, pain, fatigue, and impaired coordination. Internally, the body’s immune system is attacking its own nervous system. Existing treatments seek to contain the immune response or divert it from the central nervous system, but a new treatment seeks to restart and rebuild the patient’s immune system.

Hematopoietic stem cell transplantation (HSCT) has been used successfully for more than 30 years to treat certain kinds of cancers. The treatment uses chemotherapy to suppress the immune system. Patients are then given an infusion of their own or donor stem cells. Although it has been a successful treatment, it’s still considered unconventional and can pose serious risks. Fortunately, a new randomized clinical trial has shown that its effectiveness might rival that of approved MS drugs.

“A side-by-side comparison of this magnitude had never been done,” said Paolo Muraro, MD, neurologist, Imperial College London. “It illustrates really the power of this treatment — the level of efficacy — in a way that’s very eloquent.”

Rebuilding the Immune System

To date, only one study evaluated patients to compare HSCT to standard MS treatments. Richard Burt, MD, of Northwestern University’s Feinberg School of Medicine in Evanston, Illinois, worked with colleagues on the new trial involving 110 patients with the relapsing-remitting form of MS. These patients can go long periods without symptoms, but most of the participants had relapsed twice during the year before the study, despite receiving medication.

Half of the patients received HSCT and the other half received a new medication to replace a drug that was not working well for them. Blood was collected from the patients receiving HSCT, and they were then treated with a drug cocktail that killed most of their immune cells. Afterward, they received an infusion of their own blood (including their own stem cells) to rebuild their immune systems.

Disease progression was assessed based on patient strength, coordination and speech. After a year, 34 of the patients in the drug treatment group showed disease progression (worsening), compared to just three patients from the transplant group.

Dr. Burt recognizes that HSCT may not be the best treatment option for most MS patients. Ideal candidates would be those with relapsing-remitting MS and frequent relapses, which is roughly 15 to 20 percent of MS patients.

Although skepticism about HSCT-related infections and complications still exists among U.S. physicians, HSCT adoption in Europe is increasing, suggesting that its popularity may grow over time.