Early Duchenne MD Study Finds Drug Strengthens Musculature

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by Kara Elam |

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Duchene muscular dystrophy drug candidate

Researchers have treated a zebrafish animal model with a tyrosine kinase inhibitor to study its effect on fish musculature and strength in an experiment that shows promise for future therapeutic targets to treat Duchenne Muscular Dystrophy (DMD). The study, entitled “Dasatinib as a treatment for Duchenne muscular dystrophy,” was published in the latest edition of the Oxford Journal’s Human Molecular Genetics.

About the Study

Dr. Steve Winder, PhD, Professor of Molecular Cell Biology, Department of Biomedical Science  at the University of Sheffield, U.K., and his team chose zebrafish because this experimental animal model exhibits a typical vertebrate development pattern, and in mutant (diseased) animals, abnormal muscle function is readily observable even in the earliest stages of development. With this advantage, Dr. Winder and his team tested whether the use of dasatinib, a specific tyrosine kinase inhibitor, could treat symptoms associated with DMD. Primary study findings revealed significant improvements in the physical appearance of the zebrafish musculature and increased swimming ability, as measured by both duration and distance, in the dasatinib treated fish when compared to control animals. “These data suggest great promise for pharmacological agents … as therapeutic targets for the treatment of Duchenne muscular dystrophy,” the authors write in their study.

Currently, there is no successful treatment option to cure the symptoms of DMD, but studies such as these may pave the way to future therapies for this debilitating and fatal disease.  “With a disease like DMD, even small reductions in symptoms can have big impacts on quality of life. And whilst any one therapeutic approach is unlikely to give a 100% recovery, combinations of different therapeutic approaches … could have an increased efficacy,” the authors say. Confirmatory studies need to be undertaken, and if results are positive human trials to evaluate dasatinib may well follow.

Read More Recent News

In a new study entitled Jagged 1 Rescues the Duchenne Muscular Dystrophy Phenotype,” a team of researchers identified a single gene – Jagged1 as the critical player capable of “rescuing” Duchenne muscular dystrophy phenotype in dogs that exhibit a complete absence of dystrophin. The study, published in the journal Cell, suggest Jagged1 is a potential new target for DMD therapies in a dystrophin-independent manner. Understanding Jagged1’s role in muscle development will allow researchers to better understand the molecular mechanisms behind DMD along with disease pathogenesis.