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21 Dec 2016
A ray of hope is on the horizon for patients facing the debilitating effects of the muscle-wasting disease inclusion body myositis (IBM). University of Kansas Medical Center researchers conducting a joint study with the Medical Research Council (MRC) Centre for Neuromuscular Diseases at University College London are poised to take the next step in testing the drug, arimoclomol.

There is currently no Treatment of Inclusion Body Myositis, but research conducted by the KU/UCL partnership, published in the March 23 issue of Science Translational Medicine, revealed that the drug deserves further study on its potential to slow patients' crippling debilitation.

The most common muscle disease in people over 50, IBM is incurable and causes progressive muscle degeneration leading to severe disability, paralysis and dependency.
"IBM is a disease that no one's ever heard of until it affects them or someone in their family," said Richard Barohn, M.D., KU Medical Center Vice Chancellor for Research and chairman of the neurology department, and a co-senior author of the study.


IBM at first hampers and later severely restricts patients' everyday activities. Inflammation results in loss of finger dexterity, which progresses to total loss of hand function. Thigh muscle weakness results in falling, and most patients need a cane or walker within five years of diagnosis, then a wheelchair or scooter after 10 years. More than half of IBM patients will have difficulty swallowing, which may result in poor nutrition.

"In much way it's like the Alzheimer's of muscles," Barohn said. "It occurs in an older population, and the same types of proteins accumulate in the brains of people with Alzheimer's as accumulate in the muscles of those with IBM."

"This is a dire situation," said Mazen M. Dimachkie, M.D., neurology professor and director of the neuromuscular division at KU Medical Center, and a co-senior author of the study. "We don't have a treatment that works, but we're on the hunt to find one."

What scientists have observed so far is that muscle tissue from Inclusion Body Myositis patients contains many misfolded proteins, which Dimachkie compared to crumpled pieces of paper. "You can't write on a crumpled piece of paper," he said. But Dimachkie said arimoclomol could be the right tool to refold those proteins or eliminate them. The drug was part of a previous trial KU Medical Center was part of to determine if it was safe to give patients with amyotrophic lateral sclerosis (ALS).

"Research, unfortunately, is a slow, labor-intensive process," said Dimachkie. "It needs time, resources and funding. It requires collaboration between us, UCL and Orphazyme, the company who currently owns this experimental drug."



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