A George Washington University (GW) research team led by Henry Kaminski, MD, chair of the Department of Neurology and Meta Amalia Neumann Professor of Neurology at the GW School of Medicine and Health Sciences (SMHS), recently received a $7.8 million award from the National Institutes of Health (NIH) to establish a rare disease network for myasthenia gravis. The network, which will join 24 existing NIH Rare Diseases Clinical Research Networks, will include basic and clinical investigators, patient advocacy groups, and biotechnology and pharmaceutical companies working together to enhance therapeutic development for this rare disease.
Kaminski, a renowned expert in myasthenia gravis who has devoted decades of study to the disease, is joined on the team by fellow investigators Linda Kusner, PhD, associate research professor of pharmacology and physiology at SMHS, and Alison Hall, PhD, associate dean for research workforce development at SMHS.
“Myasthenia gravis is a chronic autoimmune disease that affects how well the nerves and muscles communicate with each other. Often patients experience extreme weakness, struggle with their vision, and are even hospitalized because of difficulty breathing,” says Kaminski. “This grant will give the researcher community the needed infrastructure to study this rare disease in order to develop new therapies.”
The grant will fund research into the underlying pathophysiology of the disease. The different subtypes of myasthenia gravis are not well understood, there are no known biomarkers, and few research labs are studying the disease. Upward of 30% of patients are treatment resistant, and all suffer from undesirable or dangerous adverse treatment side effects. The research team will focus on the differences between ocular myasthenia and general myasthenia — subtypes that produce different antibodies that begin attacking nerve and muscle communication — and individualized treatments for the 10% of myasthenia gravis patients who develop tumors triggered by the disease.
Additionally, the researchers will identify and collect biospecimens for future study and follow myasthenia gravis patients in order to identify biomarkers.
To increase the research into myasthenia gravis, the grant also will support a career enhancement component featuring training and education opportunities for scientists, physicians, and the lay public. These opportunities will not only increase the pool of young investigators focusing their careers on rare diseases, specifically myasthenia gravis, but also improve awareness of the unique needs of myasthenia gravis patients.
“The grant is not just a single project, but the establishment of a resource that will drive research for many years,” says Kaminski. “Other rare disease networks funded in the last 10 years have advanced treatments for these disorders that otherwise would have been impossible.”