New Method to Treat Multiple Sclerosis: Two Compounds Work Together

First Posted: Oct 14, 2013 08:50 AM EDT
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Scientists may have uncovered a new way to treat multiple sclerosis--at least in mice. They've found a set of compounds that boost a population of progenitor cells that can in turn repair MS-damaged nerve fibers.

MS is an autoimmune disease of the brain and spinal cord. Currently, it affects more than half a million people in both North America and Europe. Its precise triggers are unknown, but scientists have noted that certain infections and a lack of vitamin D could be risk factors. Now there may be a new way to help treat MS.

Current therapies, such as interferon beta, aim to suppress the immune attack that de-myelinates nerve fibers. Unfortunately, this is only partially effective and can have significant side effects. This new effort, though, is a complementary approach. The method aimed at restoring a population of progenitor cells called oligodendrocytes, which are cells that normally keep the myelin sheaths of nerve fibers in good repair. In theory, they could fix these coatings after MS damages them. However, oligodendrocyte numbers decline sharply in MS due to a problem with the stem-like precursor cells that produce them.

"Oligodendrocyte precursor cells (OPCs) are present during progressive phases of MS, but for unknown reasons don't mature into functional oligodendrocytes," said Luke Lairson, one of the researchers, in a news release.

The scientists used a sophisticated small-model screening laboratory and then screened about 100,000 diverse compounds for any that could induce OPCs to mature or differentiate. In the end, the researchers found several good candidates. More specifically, benztropine showed a powerful ability to prevent autoimmune disease and was also effective in treating systems after they had arisen. While on its own this compound worked about just as well as existing treatments, researchers found that it also complimented treatments.

"Adding even a suboptimal level of benztropine effectively allowed us, for example, to cut the dose of fingolimod by 90 percent--and achieve the same disease-modifying effect as a normal dose of fingolimod," said Brian Lawson, one of the researchers, in a news release.

The findings are crucial for the treatment of MS. It could actually help reduce side effects by lowering the dosage amount for others compounds. That said, a lot more research will have to be completed before this compound is tested in humans. Yet it's an important step forward for treatment.

The findings are published in the journal Nature.

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