Institute fosters collaboration in neurodegenerative research

Neurotherapeutics Institute members work together to understand disease at multiple levels

The group started informally as a monthly meeting of diverse faculty—basic biomedical researchers, physician-scientists and clinicians—who had a shared interest in neurodegenerative disease mechanisms and treatment. The lively discussions that followed were so compelling and productive that the informal group officially became the Neurotherapeutics Institute (NTI) at UMass Medical School in 2010.

Since then, members of the NTI have worked together to understand disease at multiple levels, and design forward-thinking lines of investigation to attack central questions in the field.

“This expansive and collaborative approach is allowing us to unravel disease mechanisms in novel ways,” said Melissa J. Moore, PhD, the Eleanor Eustis Farrington Chair in Cancer Research, Howard Hughes Medical Institute (HHMI) Investigator and professor of biochemistry & molecular pharmacology, and one of four co-directors of the NTI. “Since NTI researchers span such a range of disciplines and methodologies, they are able to rapidly move in new directions using almost any approach available in modern biomedical research.”

The other NTI co-directors are Marc R. Freeman, PhD, associate professor of neurobiology and an early career scientist of the HHMI; Robert Brown Jr., DPhil, MD, chair and professor of neurology; and Neil Aronin, MD, professor of medicine, cell biology and microbiology & physiological systems.

Dr. Moore launched the monthly faculty discussion group to bring together the large community of neurodegenerative researchers and clinicians—fondly nicknamed the “neurodegenerates.” Moore is best known as a leader in the field of RNA processing and metabolism, but had recently turned her interests toward the role of RNA metabolism in neurodegeneration. She approached Dr. Brown, a world leader in the field of neuromuscular diseases, especially in amyotrophic lateral sclerosis (ALS), to learn more about the field. She recognized the potential in expanding that collaboration to researchers and clinicians in many different UMMS departments and laboratories, some of whom might not have even met each other before.

Several NTI members spend 100 percent of their time performing basic biomedical research aimed at understanding nervous system function and maintenance. Many are physician-scientists who interact directly with patients, but also pursue basic and clinical research projects. Because the expertise in this group is so multi-disciplinary, discussions of disease mechanisms range from structural aspects of disease molecules, to the cell biology of certain neurons, to population studies in patients.

“We are fortunate here to have a remarkable community of highly collaborative, very catalytic grade of scientist who all want to bring their diverse specialty interests to bear to how we understand brain degenerative disorder s, like ALS” said Brown.

NTI members are well positioned to rapidly translate new knowledge into treatment. Two of the most promising approaches for intervention are RNAi-based therapeutics and gene therapy—areas in which UMMS is a world leader. NTI members can partner directly with fellow scientists in the RNA Therapeutics Institute and Gene Therapy Center on the delivery of new therapies to patients.

To communicate these new discoveries and important future questions to the scientific community and the public at large, the NTI is planning a series of research symposia where discussions focus on central questions in neurodegeneration and brain injury. The first of these meetings was held in May in Boston.

Related link:
Neurotherapeutics Institute website