The National Institute of Arthritis and Musculoskeletal and Skin Diseases has awarded UCI a 5-year, $4.2 million grant to study sporadic inclusion body myositis (sIBM), which affects aging adults causing asymmetric muscle weakness and severe disability. The disease is currently untreatable and poorly understood.
Led by principal investigator, Tahseen Mozaffar, MD, a professor of neurology and director of the Division of Neuromuscular Disorders and the UCI-MDA ALS and Neuromuscular Center at University of California Irvine's School of Medicine, the project aims to provide a detailed characterization of sIBM disease progression over a two-year period and explore biomarkers associated with the disease, along with their influence on disease behavior and progression. This study will be the largest of its kind, enrolling 150 subjects, with a planned two-year follow up, the longest duration to date.
"As the US population above the age of 65 years continues to grow, we expect the prevalence of sIBM is likely to increase. 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 goal of the project is to investigate differences in disease phenotype and disease progression in a larger cohort of subjects with sporadic inclusion body myositis subjects, including muscle pathology, and to examine if a set of blood and skeletal muscle-based biomarkers influence the disease behavior. In addition, researchers will continue to examine the distribution of Kv1.3 potassium channels in blood and muscles to see if they can potentially be targeted by pharmacological agents. S. Armando Villalta, PhD, an assistant professor in the Department of Physiology & Biophysics at UCI's School of Medicine, will conduct the immunological analyses. Bin Nan, PhD, a professor of statistics at UCI's Donald Bren School of Information & Computer Sciences, will conduct the statistical analyses.
A major barrier to clinical trials in sIBM has been the lack of a full understanding of the natural history of the disease. This funding will enable us to conduct the observational study needed to study the disease phenotype, explore factors that influence disease progression and behavior, and ultimately direct future clinical trials designed to uncover potential treatments."
Tahseen Mozaffar, MD, Professor of Neurology and Director, Division of Neuromuscular Disorders and UCI-MDA ALS and Neuromuscular Center, University of California Irvine's School of Medicine
A thirteen-site consortium of myositis treatment centers has been created and is standing ready to adopt quickly any 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."