30 June 2022

Against cancer and myodystrophy

Cancer Drug May Help Treat Muscular Dystrophy

Tatiana Matveeva, "Scientific Russia"

Canadian scientists have discovered that a drug known as a colony-stimulating factor 1 receptor inhibitor (CSF1R) helped slow the development of Duchenne muscular dystrophy in mice. The drug increases the elasticity of muscle fibers, reports University of British Columbia. The scientists' findings appeared in the journal Science Translational Medicine (Babaeijandaghi et al., Metabolic reprogramming of skeletal muscle by resident macrophages points to CSF1R inhibitors as muscular dystrophy therapeutics).

Duchenne muscular dystrophy (MDD) is a severe genetic disease that leads to progressive muscle weakness and degeneration due to impaired synthesis of the protein dystrophin, which helps to keep muscle cells intact.

Symptoms of MDD usually appear in early childhood, and with age, patients face increased loss of muscle function. As the disease progresses, many patients are forced to rely on means of transportation, such as a wheelchair, which ultimately leads to impaired heart and lung function. Despite the fact that improvements in cardiology and respiratory care have increased life expectancy in recent decades, there is currently no cure for this disease.

At first, scientists studied the role of resident macrophages — a type of leukocytes — in muscle regeneration. Further, during experiments on mice, they found that CSF1R inhibitors, which deplete resident macrophages, had an unexpected effect, making muscle fibers more resistant to the type of tissue damage caused by contraction, which is characteristic of muscular dystrophy. The drug caused a change in the type of muscle fibers in the animal's body: from damage-sensitive type IIB fibers, they became damage-resistant type IIA/IIX fibers.

"Many have heard that there are different types of muscle fibers, including fast-contracting and slow-contracting muscles. By introducing this drug, we noticed that the muscle fibers actually began to move to a slower type, which is more resistant to damage caused by muscle contractions," the authors explain. 

After making the discovery, the researchers tested the drug on mice with MDD. Within a few months of treatment, they received the first successful results. Mice treated showed a higher incidence of damage–resistant muscle fibers and were able to perform physical tasks – for example, moderate running on a treadmill - with less muscle damage than rodents who did not receive the drug.

Further research is needed to determine whether CSF1R is effective in treating MDD in humans, the researchers add.

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