|Nan Luke shared her story about living with multiple sclerosis with our governing board. Read her Story of Hope on our website.|
Multiple sclerosis occurs when a person's immune system attacks the insulation that surrounds neurons. Without the insulation, the neurons can't send signals correctly between the brain and the muscles and a person slowly loses the ability to move and function.
Stem cell scientists have been working on MS therapies that involve maturing stem cells in to the type of cells that form that insulation and transplanting them into people, or that involve finding drugs to prompt the body's own stem cells to regrow the insulation (there's more about these approaches on our Multiple Sclerosis fact sheets).
The group from Scripps, funded by an Early Translation and Training grant, are taking the second approach. They started with thousands of tiny lab dishes containing the type of stem cells that ultimately mature into the cells--called oligodendrocytes--that make up the missing insulation. They then incubated those cells with about 100,000 different molecules to see if any of those molecules induced the cells to mature into oligodendrocytes. The idea was that if the molecule prodded the cell to become oligodendrocytes in the lab dish, it might work the same trick in a person with MS.
A press release from Scripps describes the results, which were published today in Nature.
Several compounds scored well as OPC differentiation-inducers. Most were compounds of unknown activity —but one, benztropine, had been well characterized and indeed was already FDA-approved for treating Parkinson’s disease. “That was a surprise, and it meant that we could move forward relatively quickly in testing it,” said graduate student Vishal A. Deshmukh, first author of the paper who performed most of these experiments.The key here is that the drug was already approved by the Food and Drug Administration, which means that the scientists wouldn't need to go through the expensive and time consuming process of testing the drug for safety before getting FDA approval to try it in humans. That saves money, but most importantly means they might be able to move more quickly to human trials.
After discovering the drug's possible use in treating MS, the group tested it in mice that had an induced form of the disease. The Scripps press release goes on:
In these tests, benztropine showed a powerful ability to prevent autoimmune disease and also was effective in treating it after symptoms had arisen—virtually eliminating the disease’s ability to relapse. Although benztropine on its own worked about as well as existing treatments, it also showed a remarkable ability to complement these existing treatments, in particular two first-line immune-suppressant therapies, interferon-beta and fingolimod.In addition to pursuing this drug as a treatment for MS, the group is also following up on some of the other molecules that had similar properties but weren't already FDA approved.