PROVIDENCE, R.I. [Brown University] A novel potential therapy based on a natural human protein significantly slows muscle damage and improves function in mice who have the same genetic mutation as boys with the most common form of muscular dystrophy, according to a paper published online Dec. 27 in the Proceedings of the National Academy of Sciences.
Duchenne Muscular Dystrophy is a fatal genetic mutation in about one of every 3,500 boys. They are unable to produce a protein called dystrophin that keeps muscles strong. By eight years of age, the boys begin to have trouble walking. By their teens they are often in wheelchairs, and by their 20s muscle function is so degraded that they die.
"This is all aimed at getting a therapy that will meaningfully improve the condition of patients," said Justin Fallon, professor of neuroscience at Brown University and the senior author of the paper. "This is an important step along that path."
This fall, the startup company Tivorsan Pharmaceuticals licensed rights from Brown to the key protein, biglycan, hoping to bring the potential therapy through clinical trials.
Biglycan restores the muscle-strengthening presence of a protein called utrophin, which is normally prevalent only in very young children. Utrophin still exists in adults, but in fewer places and not where it can help muscular dystrophy sufferers who cannot produce dystrophin, which keeps adult muscles strong.
In experiments described in the paper, Fallon's team showed that biglycan delivered to the bloodstream draws utrophin to the cellular membranes of muscle cells. Much as utrophin does when it is present in fetuses, infants and toddlers, the protein works to help the cells build and retain their strength.
In one experiment, Fallon's team found a 50-percent reduction in "centrally nucleated" fibers in the muscle tissue of biglycan treated mice compared to untre
|Contact: David Orenstein|