Duchenne muscular dystrophy is a congenital disease that affects boys almost exclusively (“Jerry’s kids“) and leads to severe muscle dysfunction and death. Current therapies, primarily corticosteroids, are very limited in effectiveness – and introduce their own side effects that can make things worse. Now researchers at Washington University School of Medicine in St. Louis are reporting on a new technique that involves introducing nanoparticles – stuffed with rapamycin – to alleviate some of the symptoms of the disease.
Rapamycin is an immunosuppressant usually used to prevent organ rejection post transplantation, but has also been shown to aid with autophagy, or getting rid of cellular waste. The team loaded specially designed nanoparticles with rapamycin and delivered the solution into mouse models of Duchenne muscular dystrophy. Mice receiving the new treatment had a significant increase in their muscular strength along with a boost in cardiac function (as measured by volume of blood pumped). These improvements were seen in both young and old mice with the disease, pointing toward a new therapy option for anyone with Duchenne.
Washington University: Nanoparticles treat muscular dystrophy in mice