Neuroscientists target disorders of the brain and spinal cord

Neurodegenerative diseases, such as Alzheimer’s, Huntington’s and Parkinson’s, and injuries to the brain and spinal cord from trauma or stroke are among the most feared of the disorders that afflict humankind.

The economic impact of these diseases is massive, and their toll in human suffering is incalculable. According to the National Institute on Aging, the direct and indirect costs of caring for patients with Alzheimer’s disease alone are at least $100 billion per year. With a rapidly aging population, the challenges that neurodegenerative diseases pose for our society will only increase.

Decades of research in molecular and cellular biology and neurobiology have revealed a myriad of potential drug targets to slow or prevent these disorders.

In an effort to aggressively translate these findings into effective therapies, the School of Medicine has formed a new interdepartmental program in Cellular Neuroscience, Neurodegeneration and Repair (CNNR) that will accelerate the pace of Yale’s research on neurodegenerative diseases and nerve injury.

The co-directors of the new program, Pietro De Camilli, M.D., Eugene Higgins Professor of Cell Biology, and Stephen M. Strittmatter, M.D., Ph.D., Vincent Coates Professor of Neurology and of neurobiology, both members of Yale’s Kavli Institute for Neuroscience, will recruit as many as seven new scientists in a variety of disciplines to join them at CNNR and create a core to interconnect the more than 100 neuroscientists who now work across the Yale campus.

“I am very excited about this new neuroscience initiative addressing the basic mechanisms of brain function together with a number of devastating neurological diseases,” says Dean and Ensign Professor of Medicine Robert J. Alpern, M.D. “The success of the program is ensured by the extraordinary quality of its two leaders, both of whom have performed groundbreaking research in this arena.”

De Camilli says the new program will foster research on molecular and cellular aspects of nervous system function in both health and disease. De Camilli’s pioneering work on synaptic vesicles, the cellular packets that deliver neurotransmitters into the junction between nerve cells, is bringing new understanding of normal brain function while also giving insights into pathological mechanisms.

In addition to grants from federal agencies, De Camilli's research receives support from The Human Frontier Science Program Organization as well as the G. Harold and Leila Y. Mathers Charitable Foundation.

“The creation of a program focused both on the cell biology of the nervous system and on its diseases recognizes that innovative research on pathogenetic mechanisms can best thrive in an environment where basic science research is very strong,” says De Camilli, who is also a Howard Hughes Medical Institute investigator. “I am delighted to start this new initiative with Dr. Steve Strittmatter, who has already been very successful in bridging fundamental neuroscience with clinically important problems.”

Five years ago, Strittmatter identified Nogo, a protein that blocks the regeneration of axons. His laboratory’s research on this important regulator (see Advances) has suggested potential therapies for brain and spinal cord injuries, stroke and neurodegenerative disorders such as Alzheimer’s disease, amyotrophic lateral sclerosis (Lou Gehrig’s disease) and multiple sclerosis.

“Establishing a hub for cellular and molecular studies in neuroscience with interdisciplinary and interdepartmental connection holds great promise for many major research advances by Yale neuroscience,” Strittmatter says. “This program will illuminate our understanding of how nerve cells function and communicate in the brain, especially as they relate to the development of novel therapeutic approaches to neurodegenerative diseases such as neural repair.” Strittmatter’s work is supported by The Amyotrophic Lateral Sclerosis Association, the Dr. Ralph and Marian Falk Medical Research Trust and the Christopher Reeve Paralysis Foundation.

Pasko Rakic, M.D., Ph.D., the chair and Dorys McConnell Duberg Professor of Neurobiology and director of the Kavli Institute at Yale, said the decision to focus on neurodegeneration, neural repair and the neuronal basis of cognitive function and dysfunction was an outcome of many meetings and deliberations of the School of Medicine’s Basic Science Research Strategic Planning Committee.

“Research on neurodegeneration and brain repair is one of the most difficult and most noble goals of neuroscience,” Rakic says. “Modern research methods will allow new advances. We are all excited about the prospect of entering this important and fast growing area of biomedical research.”