New drug shows promise against muscle wasting disease

A new drug to treat the muscle wasting disease inclusion body myositis (IBM) reverses key symptoms in mice and is safe and well-tolerated in patients, finds a new study led by the Medical Research Council (MRC) Centre for Neuromuscular Diseases at UCL and the University of Kansas Medical Center. The study found that the new drug Arimoclomol reversed the disease's effects at the cellular level and improved muscle strength in mice. A safety trial in 24 IBM patients conducted in London and Kansas found that the drug was safe and well-tolerated. IBM is the most common muscle disease in people over 45. It is incurable and causes progressive muscle degeneration leading to severe disability, paralysis and dependency. The precise cause is unknown and there are currently no effective treatments. In this study, the research team pursued a new treatment approach based on observations that muscle tissue from IBM patients contains many misfolded proteins.
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