The National Institutes of Health (NIH) awarded a research team at George Washington University (GW) $7.8 million to launch a rare disease network for myasthenia gravis. The network, one of 25 established NIH Rare Diseases Clinical Research Networks, will include basic and clinical investigators, patient advocacy groups, and biotechnology and pharmaceutical companies collaborating to develop and advance therapeutic development for this rare disease.
The First Organized Effort of this Scale for myasthenia gravis
Representing the first organized efforts to search for improvements to myasthenia gravis, the grant will fund research into the underlying pathophysiology of the disease. There is a lack of understanding of the different subtypes of myasthenia gravis, no known biomarkers, and a scarcity of research labs studying the disease.
A chronic autoimmune neuromuscular disease, myasthenia gravis causes weakness in the skeletal muscles, which are responsible for breaking and moving parts of the body, including the arms and legs. The name is Latin and Greek in origin and means “grave, or serious muscle weakness.”
A classical symptom includes muscle weakness that worsens after periods of activity and improves after periods of rest. The disorder can affect many different muscles from the ones that control the eyelid to chewing.
With no known cure but existing therapies can control the symptoms and even can help support a relatively high quality of life.
The grantor’s focus is on the underlying pathophysiology of the disease. This is the direct result of the fact that there is very little understanding of the different subtypes of myasthenia gravis, no known biomarkers and a scarcity of research labs. 30% plus of patients are treatment-resistant and all suffer from undesirable and dangerous adverse effects as a result of treatment.
The grantor (NIH) seeks to remedy this by encouraging the grantee (George Washington) to focus on studying the difference between ocular myasthenia and general myasthenia: they produce different antibodies that begin attacking nerve and muscle communication, which often change over time—and individualized medicine approaches for the 10% of myasthenia gravis patients who develop tumors triggered by the disease. The team will collect biospecimens for future study and follow myasthenia gravis patients to find biomarkers.
Henry Kaminsky, MD, Chair of the Department of Neurology and Meta Amalia Neumann Professor of Neurology at GW School of Medicine and Health Sciences (SMHS); and a world-renowned expert in myasthenia gravis.
Linda Kusner, Ph.D., an Associate Research Professor of Pharmacology and Physiology, GW SMHS
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