Portal to Progress – NIH Leads Partnership to Speed Alzheimer’s Drug Development

A public-private partnership led by the National Institutes of Health (NIH) to transform and accelerate drug development achieved a significant milestone recently with the launch of a new Alzheimer’s ‘big-data portal’ 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 the NIH, the U.S. Food and Drug Administration, and 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 data sets. 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 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 Dr. Francis Collins, adding that this latest initiative “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.”

Developed by Sage Bionetworks, a Seattle-based nonprofit 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 multi-disciplinary 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 more than 2,000 post-mortem brain samples.

“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 Dr. Richard Hodes, director of the NIH’s National Institute on Aging (NIA). “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.”

Because no publication embargo is imposed on the use of data posted to the AMP-AD Knowledge Portal, it increases the transparency, reproducibility, and translatability of basic research discoveries, said Dr. Suzana Petanceska, the 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,” she said. “Simply stated, we can work more effectively together than separately.”

Peggy Vaughn is the senior Public Affairs specialist in the Office of Communications & Public Liaison at the National Institute on Aging.