---

Lead investigator

Budget: $100,000

Disorders: Duchenne/Becker Muscular Dystrophy ,   Myotonic Dystrophy

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

A critical, unmet clinical need is the identification of effective therapies for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). When a protein in our muscles called AMP-activated protein kinase (AMPK) is turned on, it reduces disease severity in mice with DMD and DM1. However, AMPK-activating drugs in previous muscular dystrophy studies are neither safe for long-term human use, nor particularly potent. The use of better compounds to activate AMPK, those that are safe, effective, and able to be taken orally, would greatly increase their clinical impact for DMD and DM1 patients. Here, we will employ a new strategy to target AMPK for the treatment of muscular dystrophy. Specifically, we will investigate whether stimulation of AMPK with a practical, next-generation compound improves the health of mice with DMD and DM1, which would provide a better outlook for applicability in muscular dystrophy patients. We will focus on the ability of the AMPK-activating drug to restore the proper structure and function of dystrophic muscles, as well as explore the molecular mechanisms of its action. There is no better time than the present to perform this study since this drug is in clinical trials for treating diabetes. This proposal will determine the therapeutic potential of a novel AMPK-stimulating compound for the most prevalent muscular dystrophies in children and adults in Canada.

Impact: