Leukemia Drug May Treat Duchenne Muscle Dystrophy

Drug caused significant improvement in testing on animals carrying Duchenne muscle dystrophy.

A treatment for leukemia showed promise in slowing the progression of Duchenne muscle dystrophy (DMD) during a recent study.

Duchenne muscular dystrophy occurs most commonly in males and there is no cure at this time.

An investigation of the drug dasatinib, which blocks certain chemical signals that stimulate cancer growth, showed it could turn off signals in the protein dystrogly. This protein was previously found to play a role in DMD.

In a test of dasatinib on zebrafish that carried DMB, researchers found a 40% improvement in the fish’s condition, allowing it to swim both further and longer than fish in the control group.

“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,” said lead researcher Professor Steve Winder. “There is the potential that if the drug were taken immediately upon diagnosis, the disease progression could be dramatically reduced."

So far dasatinib is cleared for clinical use, and they are hopefully in moving towards some trials of the as a treatment for DMD.

"These are encouraging findings about a unique new avenue to treating Duchenne muscular dystrophy,” said Director of Researcher of UK Muscular Dystrophy Marita Pohlschmidt, MD.

“It is a complex condition and we are of the view that it will take a combination of therapies to treat it effectively. Professor Winder's approach could complement potential therapies currently advancing through clinical trials, making them more effective.”

Researchers have begun experiments for the drug on mice, in addition to an analysis of other drugs that work similarly to dasatinib.