Lee Sweeney, director of the Penn Center for Orphan Disease Research and Therapy at the University of Pennsylvania Perelman School of Medicine in Philadelphia, was awarded an MDA research grant totaling $278,286 over a period of three years to test whether a new treatment that affects muscle calcium can slow the damage to muscle tissue in several forms of muscular dystrophy.
A common characteristic of several types of muscular dystrophy is loss of regulation of calcium within muscle tissue. Calcium plays an important role in muscle contraction, and the inability to keep its level well-regulated reduces the force the muscle can generate, and contributes to muscle degeneration. Sweeney has worked on the development and testing of a peptide (a short chain of amino acids) called CT38, which helps correct the calcium handling defect in muscle. The peptide has been shown to be safe in humans, and will now be tested in models of Duchenne muscular dystrophy, Miyoshi myopathy and myotonic muscular dystrophy.
“We will give this peptide to these mouse models of human muscular dystrophies and observe whether the muscles from these mice can generate more force over time, and if the peptide prevents eventual destruction of the skeletal muscles,” Sweeney says.
“For all of the muscular dystrophies, it is clear that there is the possibility of developing multiple drugs to go after different aspects of each disease. By developing these multidrug therapies, we hope we can move toward stopping disease progression. We feel that CT38 has the potential to be one of the drugs in our arsenal that will combat the muscular dystrophies.”
Funding for this MDA grant began August 1, 2013.