Masahiro Iwamoto, research scientist at the Children’s Hospital of Philadelphia and associate professor of pediatric orthopedics at the University of Pennsylvania School of Medicine, was awarded an MDA research grant totaling $405,000 over a period of three years to study new ways to reduce muscle degeneration.
“The active form of vitamin A, called retinoic acid, plays an important role in numerous organs, including musculoskeletal systems,” says Iwamoto. His team has recently discovered that a molecule similar to retinoic acid, called an RAR-gamma agonist, blocks two different pathologic processes in muscle: formation of bone within the muscle and muscle degeneration.
Iwamoto will now build on that work to learn more about RAR-gamma signaling and how it contributes to the repair and maintenance of skeletal muscle. He will test whether a specific RAR-gamma agonist, called R667, which has shown some promise in the lab and has been tested in humans for other conditions, has potential for treatment of muscular dystrophy in mice.
“We will determine whether these drugs can in fact block or even reverse muscle degeneration in mouse models of muscular dystrophy,” he says, potentially leading to a novel means of therapeutic intervention.
Funding for this MDA grant began Feb. 1, 2013.