Stem Cell Transplant May Enhance Severe Multiple Sclerosis Treatment

Procedure may be more effective than leading drug mitoxantrone.

For patients with severe multiple sclerosis (MS), a stem cell transplant may prove to be more effective than the commonly used drug mitoxantrone, a recent study found.

Published in the February 11, 2015 online issue of Neurology, the study included 21 MS patients whose disease-related disability worsened during the previous year despite the use of first-line treatments. The patients, who were on average 36 years of age, required the use of a cane or crutch to walk.

All patients were prescribed medications to suppress immune system activity. Twelve participants received mitoxantrone, which decreases immune system activity.

The other 9 patients had stem cells harvested from their bone marrow. Following immune system suppression, stem cells were reintroduced into their body through a vein.

The cells eventually migrated to bone marrow, which produced new cells that became immune cells.

"This process appears to reset the immune system," study author Giovanni Mancardi, MD, said in a press release. "With these results, we can speculate that stem cell treatment may profoundly affect the course of the disease."

The combination of intense immunosupression prior to the stem cell treatment was found to reduce disease activity significantly more than treatment with mitoxantrone. Patients who received stem cell transplants had 80% fewer new areas of T2 lesion brain damage than patients who received mitoxantrone.

Patients in the stem cell group had an average of 2.5 new T2 lesions compared with 8 new T2 lesions in the mitoxantrone group. In terms of the MS-associated gadolinium-enhancing lesions, none of the patients in the stem cell treatment group had a new lesion appear during the study, compared with 56% of patients in the mitoxantrone group who had at least 1 new lesion.

"More research is needed with larger numbers of patients who are randomized to receive either the stem cell transplant or an approved therapy, but it's very exciting to see that this treatment may be so superior to a current treatment for people with severe MS that is not responding well to standard treatments," Dr. Mancardi said.