MDA awarded a research grant totaling $390,000 over a period of three years to Pura Muñoz-Canoves, ICREA research professor and cell biology coordinator at Pompeu Fabra University in Barcelona. The funds will help support Muñoz-Cánoves' research into strategies aimed at reducing muscle scarring (fibrosis) in people with Duchenne (DMD) and Becker (BMD) muscular dystrophies.
Fibrosis is a hallmark of DMD and a major factor in gauging disease severity. Fibrosis also compromises the efficacy of ongoing preclinical gene- and cell-delivery therapies. No treatment exists for reversing fibrosis in DMD; nor is there a clear understanding of the mechanisms underlying fibrosis development in dystrophic muscle (muscle that is deficient in dystrophin protein).
Muñoz-Canoves and colleagues plan to test whether and how a protein called PAI-1 (plasminogen activator inhibitor-1) may regulate inflammation-driven muscle degeneration and fibrosis development in muscular dystrophy.
"The discovery of new causes of fibrosis development in dystrophic muscle will provide an opportunity for new strategies to halt disease progression," Muñoz-Canoves said. "Since muscle fibrosis also represents a major obstacle for successful engraftment of stem cells in dystrophic muscle, targeting molecules promoting fibrosis appears to be an easy-to-test alternative to improve future DMD stem cell therapies.
Funding for this MDA grant began February 1, 2012.