The National Institute on Aging (NIA) has committed an estimated $73 million over the next five years to “diversify and reinvigorate the Alzheimer’s disease drug development pipeline,” according to an NIH press release. Two new Alzheimer Centers for the Discovery of New Medicines are receiving the funding. The two centers, which are multi-institutional, are: The Open Drug Discovery Center for Alzheimer’s Disease (Open-AD), and The Indiana University School of Medicine Alzheimer’s Disease Drug Discovery center.
Alzheimer’s disease is one of healthcare’s largest areas of unmet need. A critical need exists, in particular, for disease modifying treatments, as none of the current drugs affect the underlying pathology. In addition, better diagnostics are needed as well as biomarkers for assessing response to treatment. The current world market for treatments is only about $3 billion but is expected to reach between $10 billion to $15 billion by 2026. There are currently more than 130 drugs in development for Alzheimer’s, however, many of them fail in late stages.
This injection of funding, while modest, therefore raises hopes for faster progress. “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.
The centers will provide additional infrastructure for developing research tools and technologies 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 related research.
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. This 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.