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Neurodegeneration dream team
CAMBRIDGE, Mass.—A $25 million gift from the Robert A. and Renee E. Belfer Family Foundation has brought the talents of three powerhouse neurodegeneration research institutions together to create the transformative Neurodegeneration Consortium that looks to advance the study and care of Alzheimer's and other diseases.
The consortium brings together the Picower Institute of Memory and Learning at MIT, the University of Texas MD Anderson Cancer Center and Baylor College of Medicine. Each institution will bring its own strengths to bear in a robust and multifaceted partnership that will strive toward better understanding the underlying instigators of Alzheimer's disease.
The focus of the research will extend beyond the amyloid hypothesis that attracts most of the focus in the field. The amyloid hypothesis suggests that the buildup of amyloid beta- peptide in the brain is a primary influence in the neurodegeneration associated with Alzheimer's disease.
"There appear to be multiple mechanisms at work in Alzheimer's disease … there are co-conspirators beyond amyloids in the brain," says Dr. Ronald DePinho, president of MD Anderson.
Recent research at MIT, among other institutions, has suggested that neurodegeneration, cancer and other diseases associated with aging are intimately linked. Age itself appears to be the most important risk factor, leading researchers to believe that understanding the "circuitry" of aging at the molecular level holds the key to preserving patient health and enhancing quality of life in an aging population.
The consortium will attempt to leverage its members' multidimensional backgrounds to adopt an unbiased view of the problem, hitting a "reset button" for Alzheimer 's research and attempting to apply genomic and computational biological technologies to identify new therapeutic points of attack.
Picower Institute Director Li-Huei Tsai is a prominent neuroscientist specializing in Alzheimer's research, and will serve as the lead MIT investigator. The team of investigators includes Lynda Chin, DePinho, Giulio Draetta, Ming-Kuei Jang and Philip Jones of MD Anderson; Juan Botas, Joanna Jankowsky and Hui Zheng of the Baylor College of Medicine; and Hugo Bellen and Huda Zoghbi of the Howard Hughes Medical Institute and the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Baylor College of Medicine.
The Picower Institute and Baylor College will be the leading forces behind the underpinning science and pathway development. Once targets are identified, the infrastructure for drug development will be done at MD Anderson. MD Anderson's drug development team, which possesses 200 years of combined experience, is tasked with converting the research into viable clinical drug candidates.
"This consortium combines talented people and real drug development capabilities that can be converted into proof of concept," says DePinho.
Early-stage drug development has already begun on one promising new target for myeloma and brain, breast and other cancers.
The consortium's benefactor, Belfer, has supported cancer research in the past, and when he became interested in neurodegeneration, he started by asking trusted experts what the field needed to make progress. These conversations led him to assembling something of a "dream team" of scientists with expertise in Alzheimer's disease, autism, cancer and other illnesses in a robust cross-fertilization of scientific disciplines. The investigators chosen for the consortium include individuals capable not just of making scientific discoveries, but also instigating drug development.
The $25 million gift serves as a launching point for the consortium's preliminary work. However, the gift is contingent on the consortium partners securing matching donations— another $25 million in funding by Jan. 1, 2016. About $6.5 million of this funding has already been raised.
DePinho is optimistic that the consortium's preliminary data will help to secure matching funds and reignite interest in neurodegeneration among players in the pharmaceutical industry.
"Pharma has moved away from neurodegeneration because of some of the failures of past research," he says. "We hope to re-engage pharma and take new drugs to trial. The consortium being funded by a philanthropic gift allows us to be more maverick and take risks."
"We're not repeating or competing against anything anyone else is doing," says Dr. Huda Zoghbi, director of the Duncan Neurological Research Institute.
On a more somber note, DePinho recounts the impetus for the consortium's work.
"Incidence of Alzheimer 's disease rises dramatically after age 60. There will be 1.2 billion people over age 60 in the world by 2025—we stand to see a several-fold increase in a very costly disease," he says. "We need to address this problem with a higher level of urgency because we're on a collision course with a health crisis."