Antineoplastics helped slow the progression of Duchenne muscular dystrophy in mice

Researchers at the University of British Columbia’s Biomedical Engineering Department have discovered that existing anticancer drugs have potential as a treatment for muscular dystrophy.

Duchenne muscular dystrophy (DMD) is a severe hereditary disorder that leads to progressive weakness.Image Credit: University of British Columbia

Researchers have found that the drug, known as a colony-stimulating factor 1 receptor (CSF1R) inhibitor, helps slow the progression of Duchenne muscular dystrophy in mice by increasing the elasticity of muscle fibers. ..

Survey results released today Scientific translation medicine..

“This is a class of drugs already in use in clinical trials to treat rare forms of cancer,” said Dr. Farshad Babaeijandaghi, a postdoctoral fellow at UBC and lead author of the study. “It is incredibly exciting to discover that it can serve a dual purpose as a treatment for muscular dystrophy. It shows many possibilities, and with further testing, the patient’s May help extend and improve quality of life. “

Duchenne muscular dystrophy (DMD) is a severe hereditary disease that causes progressive weakness and degeneration by destroying the protein dystrophin, which helps keep muscle cells intact. It is the most common congenital disease in Canada, affecting 1 in 3,500 men and rarely women.

Symptoms of DMD usually appear in early childhood, and patients have increased loss of muscle function as they age. As the disease progresses, many patients are forced to rely on mobility aids such as wheelchairs, which ultimately affect heart and lung function. Improvements in heart and respiratory care have increased life expectancy over the last few decades, but there is currently no cure.

Muscular dystrophy is a catastrophic disease that affects young children. Although this is not a cure, it can significantly slow the progression of the disease and allow people to move longer and leave the wheelchair. It can be used in combination with other treatments and new gene therapy approaches aimed at genetic defects. “

Professor, Dr. Fabio Rossi, Lead Author of Research, UBC Biomedical Engineering and Genetic Medicine

Researchers were amazed at this discovery when they were first studying the role of resident macrophages (a type of white blood cell) in muscle regeneration.

During experiments in mice, they have the unexpected effect that CSF1R inhibitors that deplete resident macrophages make muscle fibers more resistant to the types of contractile-induced tissue damage characteristic of muscular dystrophy. I found that. The drug was effective in changing the type of muscle fiber in the animal’s body from damage-sensitive type IIB fibers to damage-resistant type IIA / IIX fibers.

Many have heard that there are different types of muscle fibers, including fast and slow spasms. By administering this drug, we observed that the muscle fibers actually began to transition to a slower, more tolerant, spasmodic form that was more resistant to the damage caused by muscle contraction. “

Dr. Rossi

After making the discovery, the researchers tested the drug in DMD mice. Within months of treatment, they began to see successful results. Treated mice had a higher frequency of injury-resistant muscle fibers and were able to perform physical tasks such as moderate running on a treadmill with less muscle damage than untreated mice.

The result was actually very dramatic. The improvement in muscle elasticity was profound. “

Dr. AS Farshad Babaeijandaghi

Researchers say further research is needed to determine whether CSF1R is effective in treating human DMD. Given that some short-term clinical studies have already shown that this class of medicine can be safely used by people, they mean that patient-ready treatment is imminent. I’m hoping it might be.

“Developing new drugs can be a long process,” says Dr. Rossi. “But since the safety profile of this drug has already been proven in human studies, it may mean that we are on the fast path to new treatments for muscular dystrophy.”