With the growing aging population, Alzheimer’s disease is among the greatest public health challenges of the 21st century. It affects an estimated 5.6 million people age 65 and older in the U.S. alone, a number that could rise as high as 14 million by 2050 without effective treatment and prevention.

The National Institute on Aging (NIA) is awarding approximately $37 million over five years to a team of international researchers, led by Emory University, to help accelerate the development of promising new therapies that will effectively treat or prevent Alzheimer’s disease (AD). NIA is part of the National Institutes of Health.

The grant is part of a larger $73 million-dollar program by NIA to establish the Alzheimer’s Centers for the Discovery of New Medicines, two new research centers designed to diversify and reinvigorate the Alzheimer’s disease drug development pipeline.

“Drug development for Alzheimer’s disease is a challenging, costly and high-risk endeavor,” says NIA Director Richard J. Hodes, MD. “The launch of these centers marks a strategic step forward in our multi-pronged approach to accelerating discovery and development of treatments and cures for Alzheimer’s.”

The central goal of the Open-AD Center is to accelerate the development of new drugs for AD. The center will be developing research platforms, including early-stage screening of chemical compounds and other reagents for novel drug targets, that will be openly distributed for the research community to perform in vitro, preclinical and clinical evaluation across a wide range of AD therapeutic hypotheses. It is designed to test the overarching hypothesis that open drug discovery will accelerate the development of AD medicines by catalyzing robust experimental evaluation of a series of complementary therapeutic hypotheses.

“Through these centers, NIH will expand the use of open-science and open-source principles to de-risk novel drug targets with the goal of facilitating the development of new treatments for Alzheimer’s,” says NIH Director Francis S. Collins, MD, PhD.

According to researchers, there are many new data-driven discoveries being made into the complex causes of AD and related disorders. However, a major gap between the discovery of the biological mechanisms leading to AD and the development of new therapies is the lack of high-quality research tools, including chemical probes, needed to validate new drug targets and to advance them into successful development of new drugs. The new NIA program is designed to bridge that gap and allow academic and industry partners to work together to move discovery forward faster.

“Given the widespread aging of the U.S. baby boomer population, and current unsuccessful treatment approaches focused on amyloid and select other therapeutic targets, this substantial investment by NIA answers a critical and time-sensitive need for a diverse portfolio of well characterized new therapeutic and diagnostic targets for Alzheimer's disease,” says Allan Levey, MD, PhD, director of the Goizueta Alzheimer’s Disease Research Center at Emory.

With the growing aging population, Alzheimer’s disease is among the greatest public health challenges of the 21st century. It affects an estimated 5.6 million people age 65 and older in the U.S. alone, a number that could rise as high as 14 million by 2050 without effective treatment and prevention.

The Open-AD Center brings together a team of leading academic and industry partners with deep expertise in AD research and in drug discovery. The team includes investigators with experience in AD drug discovery; as well as those involved in multiple NIA-funded open science initiatives, including the Accelerating Medicines Partnership in AD (AMP- AD) Target Discovery Program, the MODEL-AD Consortium for next generation animal models of AD; and researchers leading the development of the AMP-AD Knowledge Portal and Agora platforms for open data sharing and target prioritization.

The research team also includes world-class investigators with many years of experience in chemical probe discovery and drug development in broad areas of medicine, including cancer. Haian Fu, PhD, chair of the Department of Pharmacology and Chemical Biology at Emory and a multi-PI of the grant, is leading one of the key cores of the Open-AD Center, screening new compounds as an essential first step. Fu currently directs the Emory Chemical Biology Discovery Center and co-leads the Emory initiative on Biological Discovery through Chemical Innovation.

“This unique and multi-disciplinary collaboration is indicative of the need to approach Alzheimer’s disease from many different angles and help target multiple aspects of the disease on our journey for successful treatments,” says Fu.

Other components of the Open-AD Center are led by investigators from the SGC that have pioneered an outcomes-focused open model of target validation and early drug discovery. Understanding that many drug discovery functions are best performed by industry specialists, Open-AD has partnered with several long-time industry collaborators who can provide scientific expertise, contribute in-kind resources and bring unparalleled expertise and enviable track records of delivering against milestones. These include scientists from Thermo Fisher, Charles River Labs, X-Chem and Eli Lilly.

The Goizueta Alzheimer’s Disease Research Center is part of the Emory Brain Health Center and is one of 32 Alzheimer’s disease research centers nationally, and one of only a few in the Southeast, that are supported by the National Institutes of Health. This network of Alzheimer’s disease research centers is working collaboratively to find a treatment for the disease by 2025.

This is National Institute on Aging grant number U54AG065187