March 5, 2024

A Cancer Drug Could Potentially Treat Muscular Dystrophy

” This is a class of drug that is already being utilized in scientific trials to treat unusual types of cancer,” says Dr. Farshad Babaeijandaghi, a postdoctoral fellow at UBC and first author on the study. “To discover that it might possibly serve a double purpose as a treatment for muscular dystrophy is incredibly interesting. DMD symptoms frequently establish in early infancy, with clients experiencing progressive muscular function loss as they develop.” Muscular dystrophy is a devastating illness that impacts children at a young age. The mice that went through treatment showed greater frequencies of damage-resistant muscle fibers and were able to carry out physical tasks, like moderate running on a treadmill, with less muscle damage than their without treatment equivalents.

” This is a class of drug that is already being utilized in clinical trials to treat rare kinds of cancer,” says Dr. Farshad Babaeijandaghi, a postdoctoral fellow at UBC and very first author on the study. “To find that it could potentially serve a double function as a treatment for muscular dystrophy is exceptionally interesting. It reveals a great deal of guarantee, and with further screening, might help enhance and extend quality of life for clients.”
Duchenne muscular dystrophy (DMD) is a serious hereditary condition identified by progressive muscle weakening and degeneration triggered by problems in the protein dystrophin, which assists maintain muscle cells. It is the most widespread genetic condition in Canada, affecting about one in every 3,500 young boys and, in rare events, women.
DMD signs frequently establish in early infancy, with clients experiencing progressive muscular function loss as they mature. As the condition establishes, numerous people must depend upon mobility aids such as a wheelchair, and the illness ultimately impacts heart and lung function. While advances in heart and respiratory treatment have actually improved life span in recent years, there is no treatment at the moment.
” Muscular dystrophy is a devastating disease that affects children at a young age. While this is not a cure, it could considerably delay disease progression, helping people remain mobile and out of wheelchairs for longer,” states the studys senior author Dr. Fabio Rossi, a teacher at UBCs school of biomedical engineering and department of medical genes. “It could be utilized in combination with other treatments and emerging gene treatment approaches intended at the genetic defect.”
The findings captured the researchers by surprise while they were at first studying the function of resident macrophages– a type of leukocyte– in muscle regeneration.
Throughout experimentation in mice, they found that CSF1R inhibitors, which diminish resident macrophages, had the unexpected effect of making muscle fibers more resistant to the type of contraction-induced tissue damage that is particular of muscular dystrophy. The drug had the result of changing the kind of muscle fibers in the animals body from damage-sensitive type IIB fibers to damage-resistant type IIA/IIX fibers.
” Many individuals will have heard that there are different types of muscle fibers, including fast-twitch and slow-twitch muscles. By administering this drug, we observed that the muscle fibers actually started to transition to a slower-twitch type that is more resistant to damage brought on by contraction,” says Dr. Rossi.
After making the discovery the scientists evaluated the drug in mice with DMD. Within a few months of treatment, they began to see successful outcomes. The mice that went through treatment showed higher frequencies of damage-resistant muscle fibers and had the ability to carry out physical tasks, like moderate working on a treadmill, with less muscle damage than their neglected counterparts.
” The results were actually quite dramatic. The improvement in muscle resiliency was extensive,” says Dr. Babaeijandaghi.
If CSF1R is effective at dealing with DMD in human beings, the scientists say that more studies are required to determine. Considering that a number of short-term scientific studies have currently revealed that this class of drug is safe for use in individuals, theyre confident that it might mean a patient-ready treatment is on the horizon.
” Developing a brand-new drug can be a long procedure,” states Dr. Rossi. “But with the safety profile for this drug already being shown in human research studies, it might suggest were on a quick track to a brand-new treatment for muscular dystrophy.”
Reference: “Metabolic reprogramming of skeletal muscle by resident macrophages indicate CSF1R inhibitors as muscular dystrophy rehabs” by Farshad Babaeijandaghi, Ryan Cheng, Nasim Kajabadi, Hesham Soliman, Chih-Kai Chang, Josh Smandych, Lin Wei Tung, Reece Long, Amirhossein Ghassemi and Fabio M. V. Rossi, 29 June 2022, Science Translational Medicine.DOI: 10.1126/ scitranslmed.abg7504.
The research study was funded by the Canadian Institutes of Health Research.

Muscular dystrophy is a group of illness that results in progressive muscle weakness.
A Potential Treatment for Muscular Dystrophy
The School of Biomedical Engineering at the University of British Columbia (UBC) exposed that an existing cancer treatment might be used to treat muscular dystrophy.
The drug, known as a colony-stimulating element 1 receptor (CSF1R) inhibitor, was found to considerably stop the development of Duchenne muscular dystrophy in mice by boosting the durability of muscle fibers.
The outcomes were just recently released in the journal Science Translational Medicine.