Postdoctoral research fellow Tathagata Chaudhuri, at the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, was awarded an MDA development grant (DG) totaling $180,000 over three years to develop a stem cell therapy for muscular dystrophies, including Duchenne muscular dystrophy (DMD), that will lead to muscle regeneration.
MDA development grants are awarded to exceptional postdoctoral candidates who have the best chance of becoming independent researchers and future leaders of neuromuscular disease research.
Some experimental cell therapies for muscular dystrophies are designed to use cells from healthy human donors, but the successful transplantation of cells from one person to another requires the use of immunosuppressants in order to keep the body from rejecting the new cells.
Chaudhuri and colleagues are working on the development of a cell-based therapy that uses a person's own stem cells — adult mesenchymal stem cells (MSCs), which are taken from bone marrow and coaxed into becoming muscle cells.
The team first plans to demonstrate that MSCs can reliably be differentiated (grown) into muscle cells. They'll then use the cells they produce to study muscle regeneration in animal models of muscular dystrophy.
Next, Chaudhuri and his team will determine the optimum time for transplanting the maturing cells into a recipient. They'll monitor the development of the human MSCs in animal models to determine how well the MSCs integrate with existing muscle fibers and whether they have any effect on muscle repair and regeneration over time.
Treatment with adult stem cells that develop into muscle cells may provide a means to correct skeletal muscle function in muscular dystrophy, Chaudhuri says, noting that this type of therapy for DMD and other dystrophies "is currently becoming an increasingly attractive area of research in the muscular dystrophy community."
Funding for this MDA grant began Aug. 1, 2012.