Duchenne Muscular Dystrophy Could Be Treated With Biglycan, Study Suggests

January 2, 2011 Updated: January 10, 2011

Mice with Duchenne Muscular Dystrophy (DMD) can be treated by injecting them with a natural human protein called biglycan, new research published online Dec. 27, 2010, in the Proceedings of the National Academy of Sciences found.

DMD is the most prevalent form of muscular dystrophy. It is a hereditary disease that affects 1 in 3,500 boys in the United States, according to a press release. It is much less common in girls because the recessive gene lies on the X-chromosome, so a girl would only have DMD if the gene were on both X-chromosomes, while a boy would have DMD as long as the gene is on his one X-chromosome.

The majority of DMD patients die by their mid-20s. Currently there is no known cure.

DMD is caused by mutations in the gene that produces dystrophin, a protein that connects the cytoskeleton of the muscle fibers to the extracellular matrix, maintaining muscle strength. The absence of dystrophin leads to muscle degeneration.

The new research, titled “Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in [dystrophin-deficient] mdx mice,” showed that the human protein biglycan significantly slows muscle damage in mdx mice.

Biglycan draws utrophin to the sarcolemma (muscle membrane). Utrophin is a homolog of dystrophin, meaning it is in the same family of proteins as dystrophin, and thus can take on similar functions.

“Utrophin is required for the rhBGN therapeutic effect,” the study authors stated in the abstract. “Several lines of evidence indicate that biglycan acts by recruiting utrophin protein to the muscle membrane.”

The research suggests that injecting recombinant human biglycan (rhBGN) could be a way to substitute the homolog utrophin for dystrophin, and could prove to be a potential therapy for DMD.

Throughout the testing, there was no indication of side effects of biglycan on kidney or liver function. But the experiments clearly showed biglycan improved muscle function, while slowing the degenerative effects of DMD.

In one experiment, it was found that mdx mice treated with biglycan lost their muscle strength 30 percent more slowly compared with untreated mice.

“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, according to themedguru.com.

To read the research paper, please visit http://bit.ly/dWo7nx