Research ArticleMYOPATHY

Targeting protein homeostasis in sporadic inclusion body myositis

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Science Translational Medicine  23 Mar 2016:
Vol. 8, Issue 331, pp. 331ra41
DOI: 10.1126/scitranslmed.aad4583

Targeting protein dyshomeostasis in myopathy

Sporadic inclusion body myositis (sIBM) is a debilitating adult myopathy that is difficult to treat. Although both inflammation and protein dyshomeostasis have been implicated in sIBM pathogenesis, treatments have only targeted the inflammatory component, and all have failed in clinical trials. In a new study, Ahmed et al. tested the effects of targeting protein dyshomeostasis using arimoclomol, a co-inducer of the heat shock response. In rat myoblast cell culture, arimoclomol reduced key pathological features of IBM. In mutant valosin-containing protein (VCP) mice, which develop an inclusion body myopathy, treatment with arimoclomol ameliorated disease pathology and improved muscle function. The authors then treated a small number of sIBM patients with arimoclomol and showed that it was safe and well tolerated.