New Treatment Target for Muscle Dystrophy Discovered

Dystrophic muscle shows high levels of an enzyme that impairs muscle repair.

Researchers in a recent study found that the muscle cells of patients with muscular dystrophy have high levels of elastase, an enzyme protein the impairs muscle repair.

Duchenne muscular dystrophy (DMD) is the most common and severe type of muscle dystrophy. The stem cells that repair damaged muscle are impaired in patients with DMD, according to a study published in Scientific Reports.

In the study, researchers analyzed the molecular composition of the environment of the muscle stem cells and were able to identify higher levels of neutrophil elastase in DMD muscle compared with health muscle.

Elastase is able to break down certain proteins in connective tissue in various organs, including muscles. The enzyme is created by neutrophils responsible for inflammatory and immune responses and could contribute to the development of the disease, according to the study.

Researchers were able to show that high levels of elastase decreased survival and operation of muscle stem cells.

"Our findings evidence the importance of inflammation in muscular dystrophy, and suggest that elevated levels of elastase could play a key role in the progressive muscle degeneration seen in patients affected by DMD,” said lead researcher Dada Pisconti, PhD. "Although there is no cure for muscular dystrophy, improvements in treatments could help control symptoms to improve quality of life. Our next steps are to investigate whether drugs that target elastase are effective and safe as a potential therapy for this disease."