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Nanoparticles treat muscular dystrophy in mice
Date:2/11/2014

. What is exciting about our approach is that there are existing drugs that activate autophagy. And by repackaging the drug on nanoparticles, we can target it specifically to muscles and correct the defect in the cells' ability to recycle waste."

When treated with rapamycin nanoparticles, the mice showed a 30 percent increase in grip strength and a significant improvement in cardiac function, based on an increase in the volume of blood the heart pumped.

"An important aspect of our study is that we are treating both skeletal muscle and heart muscle with the same drug," Wickline said. "The heart is a difficult organ to treat in muscular dystrophy. But even in older animals, this regimen works well to recover heart function, and it is effective over a short period of time and after only a few doses."

"Death from Duchenne in many people is due to heart dysfunction," said Weihl, who also treats patients with neuromuscular disorders at Barnes-Jewish Hospital. "So even improving cardiac function by 10 percent in late-stage disease could be very important."

The nanoparticle used in the study consists of an inert core made of perfluorocarbon, originally designed as a blood substitute. The particles are about 200 nanometers in diameter500 times smaller than the thickness of a human hair. The surface of the nanoparticle is coated with rapamycin, which suppresses the immune system. The drug typically is used to help prevent organ rejection in transplant patients. It is known for its anti-inflammatory properties and, more recently, for its role in activating autophagy.

When injected into the bloodstream, according to Wickline, the nanoparticles accumulate in areas of inflammation, where damaged tissues have leaky blood vessels and are undergoing cell death and repair.

"The nanoparticles tend to penetrate and be retained in areas of inflammation," Wickline said. "Then they release the rapamycin over a period of time, so the dr
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Contact: Julia Evangelou Strait
straitj@wustl.edu
314-286-0141
Washington University School of Medicine
Source:Eurekalert  

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