NIH-led Effort Launches Big Data Portal For Alzheimer’s Drug Discovery

Innovative collaboration, rapid data-sharing opens research to wider community

A National Institutes of Health-led public-private partnership to transform and accelerate drug development achieved a significant milestone today with the launch of a new Alzheimer’s Big Data portal — including delivery of the first wave of data — for use by the research community. The new data sharing and analysis resource is part of the Accelerating Medicines Partnership (AMP), an unprecedented venture bringing together NIH, the U.S. Food and Drug Administration, industry and academic scientists from a variety of disciplines to translate knowledge faster and more successfully into new therapies.

The opening of the AMP-AD Knowledge Portal and release of the first wave of data will enable sharing and analyses of large and complex biomedical datasets. Researchers believe this approach will ramp up the development of predictive models of Alzheimer’s disease and enable the selection of novel targets that drive the changes in molecular networks leading to the clinical signs and symptoms of the disease.

“We are determined to reduce the cost and time it takes to discover viable therapeutic targets and bring new diagnostics and effective therapies to people with Alzheimer’s. That demands a new way of doing business,” said NIH Director Francis S. Collins, M.D., Ph.D. “The AD initiative of AMP is one way we can revolutionize Alzheimer’s research and drug development by applying the principles of open science to the use and analysis of large and complex human data sets.”

Developed by Sage Bionetworks , a Seattle-based non-profit organization promoting open science, the portal will house several waves of Big Data to be generated over the five years of the AMP-AD Target Discovery and Preclinical Validation Project by multidisciplinary academic groups. The academic teams, in collaboration with Sage Bionetworks data scientists and industry bioinformatics and drug discovery experts, will work collectively to apply cutting-edge analytical approaches to integrate molecular and clinical data from over 2,000 postmortem brain samples.

The National Institute on Aging (NIA) at NIH supports and coordinates the multidisciplinary groups contributing data to the portal. The AMP Steering Committee for the Alzheimer’s Disease Project is composed of NIA and the National Institute of Neurological Disorders and Stroke, both of NIH, the U.S. Food and Drug Administration, four pharmaceutical companies (AbbVie, Biogen Idec, GlaxoSmithKline and Lilly) and four non-profit groups (Alzheimer’s Association, Alzheimer’s Drug Discovery Foundation, Geoffrey Beene Foundation and USAgainst Alzheimer’s) and is managed through the Foundation for the NIH.

“The enormous complexity of the human brain and the processes involved in development and progression of Alzheimer’s disease have been major barriers to drug development,” said NIA Director Richard J. Hodes, M.D. “Now that we are gathering the data and developing the tools needed to tackle this complexity, it is key to make them widely accessible to the research community so we can speed up the development of critically needed therapies”

The consortium of academic teams contributing the data are led by researchers at the following institutions: Eric Schadt, Ph.D., Icahn School of Medicine at Mount Sinai, New York; Philip De Jager, M.D., Ph.D., Eli and Edythe L. Broad Institute of MIT and Harvard, Boston; Todd Golde, M.D., Ph.D., University of Florida, Gainesville; and Alan Levey, M.D., Ph.D., Emory University, Atlanta. Researchers from Rush University, Chicago; Mayo Clinic, Jacksonville, Fla.; Institute for Systems Biology, Seattle; the University of California, Los Angeles and a number of other academic centers are also participating.

Because no publication embargo is imposed on the use of the data once they are posted to the AMP-AD Knowledge Portal, it increases the transparency, reproducibility and translatability of basic research discoveries, according to Suzana Petanceska, Ph.D., NIA’s program director leading the AMP-AD Target Discovery Project.

“The era of Big Data and open science can be a game-changer in our ability to choose therapeutic targets for Alzheimer’s that may lead to effective therapies tailored to diverse patients,” Petanceska said. “Simply stated, we can work more effectively together than separately.”

About AMP-AD: The Alzheimer’s disease initiative is a project of the Accelerating Medicines Partnership, a joint venture among the National Institutes of Health, the Food and Drug Administration, 10 biopharmaceutical companies and multiple non-profits, managed by the Foundation for the NIH, to identify and validate promising biological targets of disease. AMP-AD is one of the three initiatives under the AMP umbrella; the other two are focused on type 2 diabetes and the autoimmune disorders rheumatoid arthritis and systemic lupus erythematosus.

About the National Institute on Aging: The NIA leads the federal government effort conducting and supporting research on aging and the health and well-being of older people. It provides information on age-related cognitive change and neurodegenerative disease specifically at its Alzheimer’s Disease Education and Referral (ADEAR) Center at http://www.nia.nih.gov/Alzheimers.

About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.