MICA: The role of MMP inhibitors in ameliorating muscular dystrophy

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Muscular dystrophies are a set of inherited conditions that show progressive muscle wasting and loss of function such that affected individuals become wheelchair bound and suffer premature death. While medical treatment to aid the respiratory muscles and the heart both prolongs lifespan and improves quality of life, there are no treatments that prevent the inevitable progressive loss of muscle and the consequent loss of independence. The repeated cycles of muscle damage cause inflammation and scar tissue formation and this very likely inhibits effective muscle repair. This project will test the ability of a specific inhibitor of one of the enzymes associated with this scarring process to alter the disease progression in two mouse models of muscular dystrophy.The enzyme inhibitor was developed and tested in clinical trial for a different disease by AstraZeneca and so a lot is known about the safety of this drug. Therefore if it is effective in improving muscle function in the mice, it could be rapidly taken into clinical trial for muscular dystrophy avoiding the delays normally associated with the drug development process.