A drug developed in Corvallis and Perth, Australia, to treat a genetic disorder may also represent a promising advance in personalized medicine. Eteplirsen targets Duchenne muscular dystrophy, which leads to muscle degeneration and weakness. The pharmaceutical emerged from research by Patrick Iversen at Oregon State and is now being developed by Sarepta Therapeutics.
Duchenne arises from genetic mutations that stop muscles from making a crucial protein, dystrophin. Symptoms become apparent between the ages of 3 and 5, nearly always in boys. “This is a devastating disease,” says Iversen. “Few people with Duchenne live past their 20s.”
Eteplirsen works by changing the expression of the mutated gene, enabling muscles to make dystrophin. This approach, adds Iversen, a research professor in the Department of Environmental and Molecular Toxicology, could lead to treatments for many other rare genetic diseases. As of December 2015, Eteplirsen was under review by the U.S. Food and Drug Administration.