Emory Health Sciences

The National Institute on Aging (NIA), part of the National Institutes of Health, has awarded a grant expected to total $37.5 million over five years to establish the Open-AD Drug Discovery Center. Led by Emory University, the Center includes investigators at Sage Bionetworks, Structural Genomics Consortium (SGC), Stanford University, Oxford University, and University of North Carolina.

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 (AD) drug development pipeline.

The other NIA-funded center will be led by researchers from Indiana University in partnership with Purdue University.

“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, MD, PhD.

There are many new data-driven discoveries being made into the complex causes of Alzheimer’s disease and related disorders, but there is a significant lack of high-quality research tools, including selective protein inhibitors called chemical probes, to test the effectiveness of these discoveries, which hinders progress toward new therapies.

The central goal of the Open-AD Center is to accelerate the development of new drugs for AD. The Center will develop 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. Further, the research program will 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.

Throughout the drug discovery process, all output, including new molecules, will be made openly and rapidly available and without patents or any intellectual property restrictions. This novel and inclusive approach will enable scientists, clinicians, patients and families to engage in experiments without restriction on data sharing, so that results can be disseminated rapidly with anyone.

The Open-AD Center is led by co-principal investigators:

• Allan Levey, MD, PhD, Professor and Chair of the Department of Neurology at Emory University, Director of Emory’s Goizueta Alzheimer’s Disease Research Center
• Lara Mangravite, PhD, President of Sage Bionetworks
• Aled Edwards, PhD, Director of the Structural Genomics Consortium (SGC), University of Toronto
• Frank Longo, MD, PhD, Chair of the Department of Neurology and Neurological Sciences at Stanford University
• Opher Gileadi, PhD, Principal Investigator at SGC-Oxford, Nuffield Department of Medicine, University of Oxford
• Stephen Frye, PhD, Director of the Center for Integrative Chemical Biology and Drug Discovery at University of North Carolina-Chapel Hill
• Haian Fu, PhD, Chair of the Department of Pharmacology and Chemical Biology at Emory University

“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,” said Dr. Levey from Emory University.

“Open-AD builds on the existing open science infrastructure of the Accelerating Medicines Partnership in Alzheimer’s Disease target discovery program by developing and openly distributing reliable experimental reagents to advance the experimental validation of target hypotheses,” said Dr. Mangravite, President of Sage Bionetworks.

“Open distribution of tool compounds supports the thorough evaluation of targets and their mechanisms using diverse inquiries across many research teams.” said Dr. Edwards, Director of the Structural Genomics Consortium. “This approach, which has been used for more than a decade by the SGC, works to rapidly de-risk targets and to advance biological knowledge.”

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This is National Institute on Aging grant number U54AG065187.

About SGC

The SGC is a pre-competitive public-private partnership that accelerates research in human biology and drug discovery by making all of its research output freely available to the scientific community. To achieve its mission, the organization is building an open and collaborative network with hundreds of scientists from academia and industry worldwide. The SGC has active research facilities at seven leading academic institutions across the globe (Universities of Toronto and McGill Canada, University of Oxford-UK, UNICAMP-Brazil, Karolinska Institute-Sweden, UNC Chapel Hill-USA and Goethe University Frankfurt-Germany). The SGC is a registered charity (number 1097737) that receives funds from AbbVie, Bayer Pharma AG, Boehringer Ingelheim, Canada Foundation for Innovation, Eshelman Institute for Innovation, Genome Canada, Innovative Medicines Initiative (EU/EFPIA), Janssen, MSD, Merck KGaA, Novartis Pharma AG, Pfizer, São Paulo Research Foundation-FAPESP, Takeda, and Wellcome Trust. For more information, visit http://www.thesgc.org.

About Emory

The Goizueta Alzheimer’s Disease Research Center is part of Emory University School of Medicine’s Emory Brain Health Center. It 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. The Goizueta ADRC has pioneered research programs that are helping drive forward actions to support the national plan for Alzheimer’s Disease and related disorders. It conducts more than $11 million annually in industry-sponsored clinical trials, community outreach and education initiatives, federal research grants and philanthropically-funded research studies.

Sage Bionetworks

Sage Bionetworks is a nonprofit biomedical research and technology development organization that was founded in Seattle in 2009. Our focus is to develop and apply open practices to data-driven research for the advancement of human health. Our interdisciplinary team of scientists and engineers work together to provide researchers access to technology tools and scientific approaches to share data, benchmark methods, and explore collective insights, all backed by Sage’s gold-standard governance protocols and commitment to user-centered design. Sage is a 501c3 and is supported through a portfolio of competitive research grants, commercial partnerships, and philanthropic contributions.