Anti-Cancer Drugs Help Fight Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Treatment Found in Anti-Cancer Drugs

A new research study may provide good news for people with genetic disorders that cause muscle degeneration, including Duchenne muscular dystrophy (DMD). Currently, there are very few drugs available to help slow down muscle degeneration, but a study by the Children’s Hospital of Eastern Ontario (CHEO) and led by Dr. Robert Korneluk, a professor at University of Ottawa’s Faculty of Medicine and founder of the CHEO Research Institute’s Apoptosis Research Centre, shows that anti-cancer drugs may be another treatment option.

CHEO Dystrophy Researcher Dr. Robert Korneluk

The research team found that IAP-targeting drugs that are normally used to kill cancer cells also induce the repair and growth of muscle. The team was able to identify how this happens by activating specific cell signaling or communication pathways that promote the fusion of muscle cells to create new muscle fibers.

“We know of five pharmaceutical companies pursuing phase one clinical trials with specific drugs to treat cancer patients,” says Dr. Korneluk. “These anti-cancer drugs target the IAP genes, an important family of proteins related to tumor survival that were discovered by the CHEO group over 15 years ago. At that time, we were looking at the role of the IAP genes in cancer as well as in muscle disease. So it was only logical for us to explore the effectiveness of these drugs in both disease conditions.”

Different Dystrophy Types of Muscle Weakness Areas

In addition, the team found that the effects could be repeated using a growth factor normally found in the body known as TWEAK. When low TWEAK levels were introduced, the same signaling pathway was activated, which further supports muscle tissue repair.

“We think it’s reasonable to move into clinical trials with this methodology within the next couple of years,” says Eric LaCasse, CHEO associate research scientist. “Regulatory bodies need proof that the drug is safe, which the existing cancer trials will offer, and they need to see an evidence-based rationale–which we’ve worked hard to be able to announce today.”


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