Vladimir Ljubicic, a postdoctoral fellow in the department of cellular and molecular medicine at the University of Ottawa in Ontario, Canada, was awarded an MDA development grant (DG) totaling $180,000 over a period of three years for his investigation into potential therapies for Duchenne muscular dystrophy (DMD). (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.)
Scientists have shown that increasing levels of a structural protein called utrophin and properly localizing it in the cell appears to partially compensate for the missing dystrophin protein — the underlying cause of DMD.
Utrophin has been observed at higher levels in "slow-twitch" muscle fibers (which are less affected in DMD), and at lower levels in "fast-twitch" fibers.
"A critical question," Ljubicic said, "is whether the benefits associated with slow-twitch fibers in DMD are strictly dependent on the increased levels of utrophin, or on some other factor associated with the slow-twitch fiber type."
In human skeletal muscle cell cultures, and also in mouse models of DMD, Ljubicic will test several compounds to determine whether they promote development of slow-twitch fibers.
Because the compounds he plans to test already are in clinical trials for other disorders, favorable results from Ljubicic's research could accelerate the development and implementation of new therapies for DMD centered either on increased utrophin activity in the cell and/or promotion of the slow-twitch muscle-fiber type.
Funding for this MDA grant began February 1, 2012.