Noah Weisleder, associate professor of physiology and cell biology at Ohio State University in Columbus, was awarded an MDA research grant totaling $405,000 over a period of three years to study muscle repair for development of treatment for limb-girdle muscular dystrophies (LGMD).
In all muscular dystrophies, muscles are damaged during normal use. Part of that damage occurs when the outer membrane of the muscle cell tears under the force of normal muscle contraction. The cell has mechanisms to repair torn membranes, allowing the survival of these damaged cells, but these mechanisms are insufficient in LGMD and other muscular dystrophies.
“These findings suggest that a therapeutic approach that increases membrane repair capacity could have broad efficacy across a number of different muscular dystrophies in human patients,” Weisleder says.
His group has previously shown the importance of a protein called Mitsugumin 53 (MG53) in the repair process, and has demonstrated that it can be directly applied to cells and tissues to increase the repair capacity, including in a rodent model of Duchenne muscular dystrophy (DMD). He will now test this strategy in various rodent models of LGMD, to determine if this protein could be used as a therapeutic approach in humans.
“If MG53 can improve pathology in a number of different muscular dystrophies,” Weisleder says, “it is more likely that this protein therapy could be effectively moved towards clinical trials, as it would be able to treat a relatively large patient population spread across various types of muscular dystrophy.”
While such therapy would not be a definitive cure for any of the muscular dystrophies, it could provide meaningful therapy for patients until definitive treatments for the underlying causes of these diseases are available.
Funding for this MDA grant began Feb. 1, 2013.