UCI Health researchers awarded $4.2 million to study rare, debilitating muscle disorder
Untreatable disease causes muscle weakness, severe disability in older adults
April 07, 2021
UCI Health neuromuscular disorder researchers have been awarded a prestigious $4.2 million grant to study a little understood muscle disorder in aging adults that causes asymmetric muscle weakness and severe disability.
Known as sporadic inclusion body myositis (sIBM), the disease is currently untreatable.
The five-year grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases will enable researchers to develop a detailed characterization of sIBM progression and explore the influence of biomarkers on the behavior and progression the disease, said Dr. Tahseen Mozaffar, director of the UCI Health ALS and Neuromuscular Center, one of only six Muscular Dystrophy Association-designated centers in California.
The study will be the largest of its kind, enrolling 150 sIBM patients with a planned two-year follow up, the longest duration to date.
“As the U.S. population over age 65 continues to grow, we expect the prevalence of sIBM is likely to increase," said Mozaffar, the study's principal investigator and professor of neurology who leads the UCI School of Medicine's Division of Neuromuscular Disorders.
"Our hope is to gain a better understanding of this rare, yet devastating disease, so that we can design more effective and efficient clinical trials to identify potential treatments for this currently untreatable disease.”
The study will investigate differences in the disease phenotype and progression, as well as muscle pathology in a larger cohort of sIBM patients. Researchers also will explore whether blood and skeletal muscle-based biomarkers influence disease behavior. In addition, they will examine the distribution of Kv1.3 potassium channels in blood and muscle for possible targeting by pharmacological agents.
The study's immunological analyses will be conducted by S. Armando Villalta, PhD, an assistant professor in the School of Medicine's Department of Physiology & Biophysics. Statistical analyses will be led by Bin Nan, PhD, a professor of statistics at the university's Donald Bren School of Information & Computer Sciences.
“A major barrier to clinical trials in sIBM has been the lack of a full understanding of the natural history of the disease,” said Mozaffar. “This funding will enable us to conduct the observational studies needed to explore the disease phenotype, the factors that influence disease progression and behavior, and ultimately direct future clinical trials designed to uncover potential treatments.”
He said a consortium of 13 U.S. treatment centers for myositis, a group of rare conditions that cause muscle weakness and pain that gets progressively worse, is ready to act on the study's findings and participate in future clinical trials aimed at changing the course of sIBM.
Effective April 1, 2021, the award, number 1R01AR078340-01, is titled, “Influence of NT5c1A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic Inclusion Body Myositis – A prospective evaluation.”
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