NIH Awards $7.8M for Rare Disease Network to Advance Clinical Research in MG

Ana Pena, PhD avatar

by Ana Pena, PhD |

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The National Institutes of Health (NIH) has awarded $7.8 million for the launch of a rare disease network, called MGNet, to drive research in myasthenia gravis and which will be led by experts from George Washington University, Yale University, and Duke University.

The network will be part of the NIH Rare Diseases Clinical Research Networks, and bring together investigators, patient advocacy groups, and biotechnology and pharmaceutical companies to develop new treatments and enhance awareness for the disease.

Leading the team will be Henry J. Kaminski, MD, chair and professor of neurology at the George Washington School of Medicine and Health Sciences (SMHS), and an expert in MG who has focused on studying the underlying biology of the disease and the muscles affected in patients with ocular MG.

Other members in the consortium include Yale’s Kevin C. O’Connor, PhD, and Richard J. Nowak, MD, as project leaders and members of the executive steering committee, along with Linda Kusner, PhD, of George Washington University, and Jeffrey T. Guptill, MD, of Duke University.

Plans are to run MGNet on seven study sites to support different lines of research, seek insights on the underlying mechanisms of MG, provide more comprehensive knowledge on disease variability, and establish better clinical outcome measures — valuable information that can be used by investigators to accelerate clinical trial readiness.

Two important goals will be to understand better what distinguishes the different types of MG and find biomarkers for the disease. Specifically, the researchers will look deeper at the differences between ocular myasthenia and generalized myasthenia as well as investigate the use of personalized medicine to the 10% of patients who develop tumors as a consequence of MG.

The research team will also identify and collect samples from MG patients for future study and follow patients in order to identify treatment-responsive biomarkers. More than 30% of the patients are resistant to treatment and all experience undesirable or even dangerous side effects as a result of treatment.

Biomarkers could help select the best candidates for trials, improve monitoring, and provide potential therapeutic targets.

The team hopes to establish an MG-specific resource of biological samples and best practices that could be used by academics and industry to discover new targets and develop clinical trials.

In addition, MGNet will grant fellowships to young investigators, and fund pilot projects to aid further discovery in the field.

One of the pilot studies will determine whether a current cancer therapy that eliminates immune cells producing harmful antibodies might also be effective for MG, an autoimmune disease also triggered by dangerous antibodies that mistakenly attack proteins important for muscle function.

New training and educational opportunities will also be provided for scientists, physicians, and the public to increase the pool of young investigators focusing on MG and improve awareness for the disease.

“This grant will give the researcher community the needed infrastructure to study this rare disease in order to develop new therapies,” Kaminiski said in a news release.

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