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Dr. Farshad Babaeijandaghi Source UBC |
Researchers at UBC’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.
The researchers found that the drug — known as a colony-stimulating factor 1 receptor (CSF1R) inhibitor — helped slow the progress of Duchenne muscular dystrophy in mice by increasing the resiliency of muscle fibers.
The findings were published today in Science Translational Medicine.
“This is a class of drug that is already being used in clinical trials to treat rare forms of cancer,” says Dr. Farshad Babaeijandaghi, a postdoctoral fellow at UBC and first author on the study. “To find that it could potentially serve a double purpose as a treatment for muscular dystrophy is incredibly exciting. It shows a lot of promise, and with further testing, could help extend and improve quality of life for patients.”
Duchenne muscular dystrophy (DMD) is a severe genetic disorder that leads to progressive muscle weakness and degeneration due to disruptions to the protein dystrophin, which helps keep muscle cells intact. It is the most common congenital disease in Canada, affecting about one out of every 3,500 males, and in rarer cases, females.
DMD symptoms typically appear in early childhood, with patients facing increased loss of muscle function as they age. As the disease progresses, many patients are forced to rely on mobility aids, such as a wheelchair, with the disease eventually impacting heart and lung function. While improvements in cardiac and respiratory care have increased life expectancy in recent decades, there is currently no cure.