Mattia Quattrocelli, at the Center for Genetic Medicine, Northwestern University – Chicago, was awarded an MDA development grant totaling $180,000 over three years to study the effects of glucocorticoids on muscle repair and regeneration in Duchenne muscular dystrophy (DMD). The work, co-funded by the American Association of Neuromuscular & Electrodiagnostic Medicine Foundation for Research and Education (AANEM), is expected to help improve glucocorticoid-based treatment strategies in DMD.

Currently, people with DMD often are treated with chronic administration of glucocorticoids, although the mechanism by which steroids exert their effect on DMD is not well understood. Furthermore, the use of steroids in other forms of muscular dystrophy is not recommended. And chronic steroid use is associated with significant side effects including obesity, loss of bone mass, growth suppression and — paradoxically — muscle degeneration, too. 

With colleagues, Quattrocelli will examine the effects of steroids on dystrophic muscle repair and regeneration potential, and investigate the molecular circuitries linking these drugs to known pathways important in the pathology of muscular dystrophies.

Quattrocelli’s work could provide valuable insights for understanding the action of steroids with regard to the role of other genetic mutations influencing the course of muscular dystrophy. The work will address muscle repair mechanisms as potential drug targets for muscular dystrophies and could help design corticosteroid regimens to support muscle repair and boost muscle growth, while minimizing side effects.

Funding for this MDA development grant began Feb. 1, 2017.