MS Drug Combination Repairs Damaged Brain Cells, Reverses Paralysis

Topical medications that treat skin conditions prompt stem cells in the brain to repair damage.

A combination of topical medications may hold the key to reversing damage caused by multiple sclerosis (MS).

In a study published April 20, 2015 in the journal Nature, researchers sought to identify drugs capable of activating mouse and human brain stem cells. The study found the 2 most potent medications, one that currently treats athlete's foot and the other that currently treats eczema, can stimulate regeneration in damaged brain cells and can reverse paralysis in animal models of MS.

"To replace damaged cells, much of the stem cell field has focused on direct transplantation of stem cell-derived tissues for regenerative medicine, and that approach is likely to provide enormous benefit down the road," said co-senior author Paul Tesar, PhD, in a press release. "But here we asked if we could find a faster and less invasive approach by using drugs to activate native stem cells already in the adult nervous system and direct them to form new myelin. Our ultimate goal was to enhance the body's ability to repair itself."

While the new treatment approach may hold significant promise for MS patients, researchers still must formulate the topical medications for internal use to evaluate long-term efficacy and potential side effects.

The researchers were able to create large amounts of a special type of stem cell called an oligodendrocyte progenitor cell (OPC), which is normally found in the adult brain and spinal cord.

The study examined the effects of 727 previously known drugs on OPCs in the laboratory setting. The most promising medications fell into 2 specific chemical classes.

The 2 most promising drugs were miconazole, which is found in several over-the-counter antifungal treatments for conditions such as athlete's foot; and clobetasol, which is typically available via prescription for the treatment of skin conditions, including dermatitis.

During testing, both of these drugs exhibited the ability to stimulate OPCs to form new myelinating cells. When lab mice with a disease similar to MS were treated systemically with the medications, both drugs caused native OPCs to regenerate new myelin. The drugs showed a similar result when tested on human stem cells during testing.

"It was a striking reversal of disease severity in the mice," said co-senior author Robert Miller, PhD, in a press release. "The drugs that we identified are able to enhance the regenerative capacity of stem cells in the adult nervous system. This truly represents a paradigm shift in how we think about restoring function to multiple sclerosis patients."