Existing cancer drugs could have potential as a treatment for muscular dystrophy, researchers at UBS’s School of Biomedical Engineering concluded. They 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 fibres. The findings of the research were published on July 1 in Science Translational Medicine.
A postdoctoral fellow at UBC and first author of the study Dr Farshad Babaeijandaghi said, “This is a class of drug that is already being used in clinical trials to treat rare forms of cancer,” He further added, “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 an 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.
Professor, Dr Fabio Rossi, Study’s Senior Author, UBC’s School of Biomedical Engineering and Department of Medical Genetics “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. It could be used in conjunction with other treatments and emerging gene therapy approaches aimed at the genetic defect.”