McGill University researchers say new drug could treat Duchenne muscular dystrophy

A preclinical study at McGill University suggests that’s an experimental compound called K884 can potentially boost the natural repair abilities of muscle stem cells.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that causes severe muscle degeneration.

DMD, which affects about one in 5,000 boys worldwide, is a disorder that can result in wheelchair dependence through teenage years and life-threatening complications in early adulthood.

“By strengthening muscle repair rather than just slowing degeneration, therapies that stimulate muscle stem cell function have the potential to improve quality of life for DMD patients. It may help restore muscle function and, ultimately, offer greater independence,” said senior author Natasha Chang, an assistant professor in McGill’s department of biochemistry.

This preclinical study marks the first time the drug has been tested in DMD cells. Originally the biotechnology company Kanyr Pharma developed the drug for cancer and metabolic diseases.

Researchers analyzed DMD-affected muscle stem cells from both human and mice under a microscope to understand how to drug worked. The outcome showed that the experimental drug blocks specific enzymes, allowing muscle stem cells to develop into functional muscle tissue.

“What makes K884 particularly promising is its precision. It targets DMD-affected cells without affecting healthy muscle stem cells,” said Chang.

Unlike gene therapy, K884 works on a cellular level instead of targeting specific genetic mutation, making K884 a potential treatment option for all DMD patients, explained Chang.

The findings, published in Life Science Alliance, challenge previous assumptions of DMD and add a new lens towards the potential solution.

“This disease has historically been seen as a muscle problem caused by a missing protein called dystrophin,” said Chang. “But new research, including our own, shows that restoring stem cell function is just as critical for repairing muscle.”

The researchers plan on continuing to test the drug to understand long-term effects and safety concerns, while testing other related compounds.