Scientists have discovered a potential drug candidate that may help treat inherited muscular wasting disease.
The compound called pentamidine carries approval of the U.S. Food and Drug Administration for treating a severe type of pneumonia in people with weakened immune systems, as well as leishmaniasis, sleeping sickness and some yeast infections.
The new study from University of Oregon and the University of Rochester School of Medicine and Dentistry in New York, showed that pentamidine, when tested in genetically altered mice, counters genetic splicing defects in RNA that lead to type 1 myotonic dystrophy- one of nine types of muscular dystrophy - also known as DM1 and Steinart's disease.
"The fact that a very small library of compounds yielded a molecule capable of reversing the splicing defects associated with DM1 in both cell and mouse DM1 models suggests that a small molecule strategy could lead to a drug for this disease," said UO chemist J. Andrew Berglund.
In a separate commentary in PNAS, Thomas A. Cooper of the Baylor College of Medicine in Houston hailed the findings, noting that the compound is the first to show such promise of reversing splicing defects.
He noted that such a therapeutic approach is attractive because of the potential benefits to multiple organs affected by the disease.
Berglund added that pentamidine is an exciting lead compound because it is relatively easy to chemically modify, and hopefully one of these modified compounds could lead to a safe, long-term treatment in the future.