Scientists Show Drug Can Counteract Muscular Dystrophy In Mice

Source: NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases
Date: October 4, 2006

Summary:

Scientists have demonstrated for the first time that a single drug can rebuild damaged muscle in two strains of mice that develop diseases comparable to two human forms of muscular dystrophy. The scientists tested the drug, trichostatin A (TSA), an enzyme inhibitor, in two mouse models of muscular dystrophy (MD): one that naturally develops a disease similar to Duchenne muscular dystrophy in humans, the other genetically altered to develop a form of dystrophy similar to the human limb-girdle muscular dystrophy.

The scientists say this is the first time a drug has counteracted MD in a mouse model. However, they emphasize that the drug is only promoting muscle regeneration – it is not curing the defect that causes muscle deterioration. Further studies are needed to determine how long the drug works and if it works in larger animals with bigger muscles, such as dogs, before such drugs can be tested in people.

Commentary: Hopefully this drug will be effective in promoting muscular regeneration in humans with muscular dystrophy, and advance the field of regenerative medicine. Maybe this study will also give new momentum to research into genetic and other factors that cause muscle deterioration, and reverse this progressive disease.