An Existing Cancer Drug Shows Potential To Treat Muscular Dystrophy

Researchers at the University of British Columbia's School of Biomedical Engineering found that the drug called a colony-stimulating factor 1 receptor (CSF1R) inhibitor helped slow the progression of Duchenne muscular dystrophy (DMD) in mice by increasing the resiliency of muscle fibers.

A severe genetic disorder, Duchenne muscular dystrophy causes progressive muscle weakness and degeneration due to disruptions of the protein dystrophin, which helps keep muscle cells intact. It affects one out of every 3,500 males in Canada, and in rarer cases, females.

  “Muscular dystrophy is a devastating disease that impacts children at a young age. While this is not a cure, it could significantly delay disease progression, helping people stay mobile and out of wheelchairs for longer,” says the study’s senior author Dr. Fabio Rossi, a professor at UBC’s school of biomedical engineering and department of medical genetics. “It could be used in conjunction with other treatments and emerging gene therapy approaches aimed at the genetic defect.”

The drug was tested on mice with DMD after the discovery was made by researchers. Within a few months, they saw successful results.

The mice that were treated exhibited greater frequency of damage-resistant muscle fibres and were able to carry out physical activities, such as moderate running on a treadmill, with less muscle damage than their counterparts who weren't treated.

To determine whether CSF1R can be used to treat DMD in humans, further studies are needed. A number of short-term clinical studies have already shown that this class of drug is safe for use in humans, so they are hopeful that a patient-ready treatment may be on the horizon.

“Developing a new drug can be a long process,” says Dr. Rossi. “But with the safety profile for this drug already being proven in human studies, it could mean we’re on a fast track to a new treatment for muscular dystrophy.”