MDA awarded a grant totaling $375,000 to Louis Kunkel, director of the program in genomics at Children's Hospital in Boston, for research into compounds already approved for human use that may alter disease progression in Duchenne muscular dystrophy (DMD).
"There are thousands of compounds developed for use in humans for a variety purposes such as pain, diabetes and heart disease," Kunkel said. "The vast majority of these compounds have never been tested for their possible use in other diseases such as muscular dystrophy, something that can be easily accomplished in zebra fish."
In previous studies, Kunkel's lab has identified several compounds that increase the survival rate in zebra fish with a DMD-like disease and that are approved by the U.S. Food and Drug Administration for use in humans.
The team now plans to search for additional compounds, which it will test in the zebra fish model. The investigators will analyze indicators including survival rates, muscle function and toxicity, to determine the best candidate to go forward into mouse models.
"The beauty of the zebra fish is that they are permeable," Kunkel said. "They present an opportunity to directly test compounds in a live organism with the disease due to their large numbers, small size, transparency and their ability to absorb chemicals from their water environment."
Funding for this MDA grant began August 1, 2010.