Breakthrough in treatment of muscular dystrophy

Edmonton, AB A new drug cocktail used by a researcher in the Faculty of Medicine & Dentistry at the University of Alberta improved Duchenne muscular dystrophy symptoms in non-human lab models.

Duchenne muscular dystrophy is one a common genetic disorders caused by a lack of dystrophin, a muscle-supporting protein. The drug cocktail improved normal functioning of the mutated gene that triggers the condition in the lab. The lab models produced 10 per cent to 15 per cent normal levels of dystrophin after the therapy.

The study’s principal investigator, Toshifumi Yokota, published his findings in the peer-reviewed journal, Proceedings of the National Academy of Sciences on August 6. Targeting the “hot spot” of the gene mutation, the drug combination makes the condition less severe. Yokota has been working on this research for over five years and is now using this drug cocktail on human cells with Duchenne muscular dystrophy and hopes to see similar results.

A researcher in the Department of Medical Genetics, he holds two research chairs, The Friends of Garrett Cumming Research Chair, Muscular Dystrophy Canada; and the H.M. Toupin Neurological Science Chair.

Funded by the Neurological and Psychiatric Disorders of the National Center of Neurology and Psychiatry; the Ministry of Health, Labour and Welfare of Japan; the Foundation to Eradicate Duchenne; the U.S. Department of Defense; the National Institutes of Health; the Muscular Dystrophy Association; The Friends of Garrett Cumming Research; H.M. Toupin Neurological Science Research and Muscular Dystrophy Canada, Yokota worked with researchers in the United States and Japan.