NIH Grants $7.8M to Support Launch of Rare Disease Network for Myasthenia Gravis

NIH Grants $7.8M to Support Launch of Rare Disease Network for Myasthenia Gravis

The National Institutes of Health (NIH) awarded $7.8 million to researchers at George Washington University to launch a network for improving research, treatment, and education in myasthenia gravis.

This will be the first large and organized effort to focus on the disorder, which affects 20 in 100,000 people worldwide, and will include basic and clinical investigators, patient advocacy groups, and biotechnology and pharma companies.

The myasthenia gravis network will be led by researchers at the GW School of Medicine and Health Sciences: Henry Kaminski, MD, chair of the department of neurology, in collaboration with Linda Kusner, PhD, who is an associate research professor of pharmacology and physiology, and Alison Hall, PhD, who is associate dean for research workforce development.

Together, they will promote the myasthenia gravis network, which will be part of the 25 established NIH Rare Diseases Clinical Research Networks.

“The grant is not just a single project, but the establishment of a resource that will drive research for many years,” Kaminski said in a news release. “Other rare disease networks funded in the last 10 years have advanced treatments for these disorders that otherwise would have been impossible.”

The NIH grant will support research projects that focus on trying to understand the underlying mechanisms that trigger and mediate myasthenia gravis.

In particular, the grant aims to fund studies that will provide new insights on how to differentiate ocular and general myasthenia gravis, and to address potential strategies for individualized medicine to treat people with myasthenia gravis who also develop tumors due to the disease.

The research team will also work to select and collect biological samples from patients, which will be used in future studies to identify new biomarkers that may help monitor the disease.

In addition, the funding will support the launch of a pilot clinical study to explore the potential of anti-cancer therapies to destroy cells that are responsible for producing potentially harmful antibodies in myasthenia gravis.

“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,” 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 also support a career enhancement program, potentially result in more labs investigating myasthenia gravis, and aid further discovery in the field. The career program will include training and educational opportunities for scientists, physicians, and the general public.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.