This is the first large organized effort to search for improvements in research, treatment and education for myasthenia gravis, a chronic autoimmune disease.
The network, which will be part of 25 established 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.
Henry Kaminski, chair of the Department of Neurology and Meta Amalia Neumann Professor of Neurology at the School of Medicine and Health Sciences, is a world-renowned expert in myasthenia gravis and has spent decades studying the disease.
“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,” Dr. Kaminski said. “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 disordered physiological processes associated with 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. Upwards of 30 percent of patients are treatment resistant and all suffer from undesirable and even dangerous adverse effects as a result of treatment.
The research team will study individualized medicine approaches for the 10 percent of myasthenia gravis patients who develop tumors triggered by the disease. They will also identify and collect biospecimens for future study and follow myasthenia gravis patients in order to identify biomarkers.
Through the grant, the research team also will pilot their own clinical trial to determine whether a current cancer drug that works to eliminate cells producing harmful antibodies might also be effective for myasthenia gravis patients.
The grant also will support a career enhancement program in order to increase the number of labs researching myasthenia gravis and aid further discovery in the field.
This will include new training and educational opportunities for scientists, physicians and the lay public.
“The grant is not a just a single project, but the establishment of a resource that will drive research for many years,” Dr. Kaminski said. “Other rare disease networks funded in the last 10 years have advanced treatments for these disorders that otherwise would have been impossible.”