Human protein slows muscle damage in muscular dystrophy mice

WASHINGTON - A new study has found that 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.

Boys suffering from Duchenne Muscular Dystrophy are unable to produce a protein called dystrophin that keeps muscles strong.

“This (the study) is 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,” he added.

The key protein 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 foetuses, infants and toddlers, the protein works to help the cells build and retain their strength.

In more recent tests using an improved formulation of biglycan, Fallon said the team has seen that figure rise to 50 percent in some muscles, meaning that mice treated with biglycan are holding on to more of their function for a longer time.

He said the effects of treatment with biglycan lasted through months of testing.

Several basic tests for side effects during that timeframe, such as kidney and liver function, did not indicate any harm from the therapy.

With the efficacy of biglycan apparent in the mouse model of Duchenne Muscular Dystrophy, the researchers are eager to see if it can improve the lives of thousands of children.

“The next big step is testing in humans,” said Fallon.

The study is currently published online in the Proceedings of the National Academy of Sciences. (ANI)