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Biological Chemistry

Possible New Use For Statins

Drug Discovery: Cholesterol-lowering compounds improve muscle function in mice with Duchenne muscular dystrophy

by Michael Torrice
October 5, 2015 | A version of this story appeared in Volume 93, Issue 39

A study suggests that statins, the popular cholesterol-lowering drugs, could help children with Duchenne muscular dystrophy (DMD), a rare but lethal muscle degeneration disorder. Researchers at the University of Washington report that simvastatin (Zocor) significantly improves muscle function in a mouse model of the disease (Proc. Natl. Acad. Sci. USA 2015, DOI: 10.1073/pnas.1509536112). The compound’s possible therapeutic effects for muscular dystrophy aren’t due to its knack at reducing cholesterol levels. Instead, the researchers think statins could slow the progression of the disease through their known ability to reduce inflammation, oxidative stress, and fibrosis, a form of scarring. Nicholas P. Whitehead and his colleagues gave the drug to mice with DMD, starting the treatment at three points along the disease’s progression—before symptoms started, at an early stage, and at a late stage. The drug showed therapeutic effects at each point. Compared with untreated mice, simvastatin-treated animals had an 85% lower level of creatine kinase, a marker for muscle damage; exerted 40% more force in their hind-limb muscles; and showed 50% less fibrosis in their diaphragms.


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