Could a leukemia drug slow Duchenne Muscular Dystrophy?

A drug commonly used to treat leukemia is showing potential as a treatment that could slow the progression of Duchenne muscular dystrophy, a deadly disease that causes muscles to waste away. The condition typically affects boys and most patients don’t live past the age of 30.

For a new study, published in the journal Human Molecular Genetics, researchers investigated the drug dasatinib, which works by blocking certain chemical signals that stimulate the growth of cancer cells. They found that the same drug also switches off similar signals in a protein implicated in Duchenne Muscular Dystrophy (DMD). The protein, called dystroglycan, has a part to play in maintaining healthy muscle tissue.

The team tested the drug in zebrafish bred to carry DMD and recorded a 40 percent improvement in the condition of the fish. The fish treated with dasatinib were able to swim further and for longer than those in a control group.

It could be that by combining the drug with other treatments currently under development, their effectiveness could be improved even further.

“Dasatinib clearly has promise as a treatment for DMD,” says Steve Winder, a professor at the University of Sheffield.

“From our understanding of how the drug works we believe it could be effective in slowing muscle deterioration, prolonging patients’ ability to walk and also protecting their heart and respiratory muscles. There is the potential that if the drug were taken immediately upon diagnosis, the disease progression could be dramatically reduced.”

Because dasatinib is already cleared for clinical use, researchers hope that progress can be made more quickly toward testing the drug in human clinical trials. Experiments have already begun in mice, with promising results. Other drugs that work in a similar way to dasatinib are also under investigation.

Muscular Dystrophy UK, the Medical Research Council, and the Duchenne Parent Project NL funded the work.