New cancer drugs have ability to treat muscular dystrophy, study shows

These drugs, called histone deacetylase inhibitors, are still under development. The report examined the potential use of the drugs in promoting regeneration and repair of dystrophic muscles, thereby countering the progression of the disease, in two different mouse models of muscular dystrophy.

Led by the Burnham Institute, the study also involved the Dulbecco Telethon Institute of Rome and the National Institutes of Health.

Burnham Institute discovered that ongoing treatment with the deacetylase inhibitor Trichostatin A, currently under clinical study for breast cancer, restored skeletal muscle mass and prevented the impaired function characteristic of muscular dystrophies. Importantly, these restored muscles showed an increased resistance to contraction-coupled degeneration, the primary mechanism by which muscle function declines in muscular dystrophy.

Furthermore, muscles examined from dystrophic mice treated with Trichostatin A for three months displayed normal tissue, as compared to the muscles examined from untreated, dystrophic mice. Furthermore, dystrophic mice receiving treatment were able to perform physical exercise similar to normal, non-dystrophic mice.

“We have identified a new rationale for treating muscular dystrophy, aimed at correcting the devastating effects of a single flawed gene. This is a significant advance over the use of steroids, currently the only treatment available, which offers palliative relief, often with severe side effects,” said Lorenzo Puri assistant professor of the Burnham Institute. “Our future studies will focus on understanding precisely how several existing deacetylase inhibitors effect muscle regeneration. We will use this information to identify new compounds with similar or even better efficacy in treating muscular dystrophies.”

The Institute also commented that the results would require extensive research to determine the effectiveness of the treatment and its future availability for muscular dystrophy sufferers.

Muscular dystrophy is a group of more than 30 genetic diseases, characterized by progressive weakness and deterioration of skeletal muscles. All are inherited, caused by a mutation in one of a group of genes responsible for maintaining muscle integrity.

The study focused on the most common form, Duchenne muscular dystrophy, which affects one in 3,500 male births, according to the US National Institute of Neurological Diseases and Stroke. Inheritance is linked to the X chromosome and recessive, so the disease primarily affects boys. Most children with Duchenne muscular dystrophy die in their late teens or early 20s. The disease currently has no cure.