New translational research centers to speed, diversify Alzheimer’s drug discovery

To help meet the urgent need for therapies that will effectively treat or prevent Alzheimer’s disease, the National Institute on Aging (NIA) has launched two new research centers with funding expected to total more than $73 million over the next five years. The Alzheimer Centers for the Discovery of New Medicines are designed to diversify and reinvigorate the Alzheimer’s disease drug development pipeline. NIA is part of the National Institutes of Health.

The centers will provide added infrastructure for developing high-quality research tools and technologies needed to validate and advance the next generation of drug targets for Alzheimer’s disease. Data, research methodologies, and computational and experimental tools will be disseminated openly and free-of-charge to the broader research community—including academia and industry—for use in drug discovery and in research to better understand the complex biology of the disease.

The Accelerating Medicines Partnership-Alzheimer’s Disease (AMP-AD) program’s open-science enterprise, which has provided more than 500 new candidate targets for Alzheimer’s disease, served as the foundation for the new centers.

“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,” said NIH Director Francis S. Collins, M.D., Ph.D.

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. There are few current treatments approved by the U.S. Food and Drug Administration, and several recent, late-stage clinical trials testing disease-modifying drug candidates have failed.

“Drug development for Alzheimer’s disease is a challenging, costly and high-risk endeavor,” said NIA Director Richard J. Hodes, M.D. “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.”

Two Centers, One Mission

The Alzheimer Centers for the Discovery of New Medicines grants were awarded to two multi-institutional research teams with extensive experience in developing and promoting open-access science practices. Each team brings together world-class expertise in data science, computational biology, disease biology, structural biology, assay development, medicinal chemistry, pharmacology and clinical science.

The Open Drug Discovery Center for Alzheimer’s Disease (Open-AD) will be led by Allan Levey, M.D., Ph.D., Emory University, Atlanta; Lara Mangravite, Ph.D., Sage Bionetworks, Seattle; and Aled Edwards, Ph.D., Structural Genomics Consortium, which has research sites in North Carolina, Toronto and Oxford, UK. The research team will leverage the data and results from the AMP-AD program and develop a series of new therapeutic hypotheses centered around a prioritized set of novel targets. Open-AD will develop a suite of target enabling tools including high quality antibodies and chemical probes, and openly disseminate all data, methods and reagents to any interested academic and/or commercial investigator to accelerate validation of novel drug targets and to seed new drug discovery efforts.

The Indiana University School of Medicine Alzheimer’s Disease Drug Discovery center will be led by Alan Palkowitz, Ph.D., and Bruce Lamb, Ph.D., at Indiana University, Indianapolis, with researchers from Purdue University, West Lafayette. The center will bridge target discovery work done by the AMP-AD program with newly discovered molecules that will be studied for disease-modifying potential in Alzheimer’s disease animal models, specifically those based on human pathology, genetics and translational biomarkers developed by the NIA-supported Model Organism Development & Evaluation for Late-Onset Alzheimer’s Disease Consortium. The research team will create a diverse portfolio of Alzheimer’s disease drug targets representing new therapeutic hypotheses with a particular focus on immune pathways. Consistent with the NIH open science mission, the new center will make data and research tools available to the scientific community through an open access data sharing platform and target enablement packages.

“Through these centers, scientists will advance drug discovery for new targets to the point of attracting external partners who can take them into clinical development. Ultimately, we need many candidate therapeutics that target multiple aspects of the disease in the drug development pipeline because there’s not likely to be a single cure for Alzheimer’s,” said Lorenzo Refolo, Ph.D., program director for Alzheimer’s Translational Research at NIA.

The Alzheimer Centers for the Discovery of New Medicines are funded through NIA grant numbers U54AG065187 and U54AG065181.

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