Key Advance Toward Treatment For Most Common Adult Form Of Muscular Dystrophy - Journal Of The American Chemical Society

Main Category: Muscular Dystrophy / ALS
Article Date: 25 Nov 2008 - 4:00 PST

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Scientists in New York are reporting a critical first step toward development of a long-sought drug to treat myotonic muscular dystrophy (MMD), the most common form of muscular dystrophy in adults. MMD affects about 1 in 8,000 people. Their findings appeared in the November 8 issue of ACS' weekly Journal of the American Chemical Society.

In the study, Benjamin Miller and colleagues point out that MMD differs from typical hereditary diseases. They result from mutated DNA in genes that encodes an erroneous message that RNA picks up and passes along. As a result, cells produce faulty proteins. Those proteins disrupt cells' activity and cause symptoms of the disease. Rather, MMD is caused by wayward or "toxic" strands of RNA.

The researchers describe discovery of a family of drug-like molecules that target the errant strands of RNA, preventing production of the defective protein. The discovery, they said, provides scientists for the first time with substances that target the root cause of MMD and represent molecules that could be developed into drugs. They note that drugs more commonly target DNA or proteins, with the RNA approach offering a different and potentially valuable route to developing new medications for certain diseases.

"Dynamic combinatorial selection of molecules capable of Inhibiting the (CUG) Repeat RNA - MBNL1 interaction in vitro: Discovery of lead compounds targeting myotonic dystrophy (DM1)"
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Journal of the American Chemical Society

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Michael Bernstein
American Chemical Society