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New drug shows promise against muscle wasting disease

April 05, 2016

The Physicians’ Clinic’s Professor Mike Hanna is the senior author on a new paper that introduces a very promising drug in the treatment of the muscle wasting disease inclusion body myositis (IBM). A new study, led by the MRC Centre for Neuromuscular Diseases at University College London (UCL) and the University of Kansas Medical Center, finds that the new drug reverses key symptoms in mice and is safe and well-tolerated in patients.

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.

The study, published in Science Translational Medicine, 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.

In this study, the research team pursued a new treatment approach based on observations that muscle tissue from IBM patients contains many misfolded proteins. The research team reported results using an integrated investigational plan of the new drug, Arimoclomol, to clear these proteins out by either refolding or eliminating them.

The team started by creating cells, in a petri dish, that mimic the muscle tissue of IBM patients, and successfully tested Arimoclomol on these cells. They then used genetically modified mice whose muscle cells and symptoms closely resembled the human disease. An Arimoclomol trial in these mice found that it was well-tolerated, reversed key features of the disease, and importantly, improved muscle strength.

Following a successful patient safety trial, there are plans to begin a full-scale randomised, controlled clinical trial to formally assess if the drug is effective in slowing disease progression in people with IBM.

Director of the MRC Centre for Neuromuscular Diseases and the UCL Institute of Neurology Professor Michael Hanna, co-senior author of the paper, said, “This is an excellent example of interdisciplinary collaborative translational research that spans discovery, preclinical science and experimental medicine by a group of academic investigators supported by the MRC Centre. Targeting proteostasis has important potential benefit for patients with this disabling degenerative muscle disease. I want to congratulate the entire team and especially the MRC Centre-supported PhD students Mhoriam Ahmed, Pedro Machado, Adrian Miller and Charlotte Spicer. This work was underpinned by our critical collaboration with Linda Greensmith.”

For further information please visit http://www.mrc.ac.uk/news/browse/new-drug-shows-promise-against-muscle-wasting-disease/

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