The report shed light on the potential use of these drugs, called histone deacetylase inhibitors, in promoting regeneration and repair of dystrophic muscles, thereby countering the progression of the disease, in two different mouse models of muscular dystrophy.
Burnham Institute assistant professor Lorenzo Puri, M.D., Ph.D and his team 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 Duchenne muscular dystrophy and related dystrophies.
Indeed, muscles examined from dystrophic mice treated with Trichostatin A for three months displayed normal tissue architecture, as compared to the muscles examined from untreated, dystrophic mice. Furthermore, dystrophic mice receiving treatment were able to perform physical exercise (e.g. running on a treadmill) similar to normal, non-dystrophic mice.
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.
"We have identified a new rationale for treating muscular dystrophy, aimed at correcting the devastating effects of a single flawed gene," said Puri. "This is a significant advance over the use of steroids - currently the only treatment available - which offers palliative relief, often with severe side effects."
MEDICA.de; Source: Burnham Institute