MDA awarded a research grant totaling $390,000 over a period of three years to David Thomas, the William F. Dietrich Professor of Structural Biology and Biophysics at the Minnesota Muscle Laboratory, University of Minnesota in Minneapolis.
Defects in the sarcolemma (the membrane that surrounds a muscle fiber) of people with muscular dystrophy lead to elevated concentrations of calcium in the muscle fiber — a condition that can cause cell death.
Recent reports have indicated that increasing activity in skeletal muscle of a protein called SERCA increases calcium transport out of the fiber, lessening disease severity in mouse models of muscular dystrophy. Such an approach relies on gene therapy.
Thomas and colleagues plan to develop a small-molecule approach to achieve the same end. The group intends to identify small-molecule compounds that directly activate SERCA from skeletal muscle and then evaluate each compound's ability to restore health and function to single skeletal muscle cells and intact skeletal muscles. Finally, the investigators plan to begin testing the most promising compounds in mice.
The results of this project are expected to provide proof-of-concept of for a small-molecule strategy to treat muscular dystrophy and identify a set of lead compounds for drug development.
Funding for this MDA grant began February 1, 2012.