Alzheimer’s Disease

Overview

The Accelerating Medicines Partnership (AMP) is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private organizations. Managed through the Foundation for the NIH (FNIH), AMP aims to identify and validate the most promising biological targets for therapeutics for a range of diseases that are part of the program.

The AMP Alzheimer’s disease program is using a precision medicine approach to discover novel targets and biomarkers. The first AMP Alzheimer’s program (AD 1.0), launched in 2014, focused on discovering new therapeutic targets and evaluating the usefulness of tau imaging as a biomarker for disease progression and treatment response.  The second iteration of the AMP AD program (AMP AD 2.0) launched in March 2021 is an expansion of the initial AMP AD target discovery efforts and aims to enable a precision medicine approach to the discovery of novel targets and biomarkers.

About Alzheimer’s Disease

Alzheimer’s is a progressive brain disorder that slowly destroys memory and thinking skills. Alzheimer’s is characterized by the presence of two signature brain lesions: plaque deposits between nerve cells composed of fragments of the protein, amyloid beta (Aβ), and neurofibrillary tangles (NFT) composed of aggregated tau proteins in the interior of cells. It is the most common diagnosis for patients with dementia and the sixth leading cause of death for Americans. Experts estimate that as many as 5.8 million Americans 65 and older have Alzheimer’s disease dementia, and the prevalence in the United States is projected to increase to 13.8 million by 2050.

The financial toll of dementia is staggering; an analysis conducted by NIH-supported researchers found that total health care spending for a person with probable dementia in the last five years of life was an estimated $287,000.

Need for New Therapies

The evidence linking Aβ plaque accumulation as the cause of AD has resulted in the development of therapies by many biopharmaceutical companies. However, none to date has demonstrated clinical efficacy in patient trials. These failures may reflect problems with specific molecules and/or trial design rather than the underlying hypothesis. There is a pressing need for improved tools to support target validation in patients prior to Phase III clinical trials and to identify new targets that provide alternative approaches to targeting the disease process. Additionally, it is critical to identify reliable biomarkers that are predictive of clinical response to therapeutic intervention.

AMP Approach

Despite a substantial research and development investment for Alzheimer's disease and related dementias, and advances in our understanding of the disease pathogenesis, safe and effective treatments are still lacking. The lack of treatments reflects the need to change how the academic, biopharmaceutical, and government sectors that participate in Alzheimer’s disease research and therapy development generate, share, and use knowledge to propel therapeutic development. Real progress in developing effective therapies requires transforming the Alzheimer’s research and drug development process into one that is participatory, collaborative, well-integrated, and iterative. The AMP AD program aims to achieve this through a precompetitive partnership among government, industry, and nonprofit organizations that focuses on discovering novel, clinically relevant therapeutic targets and on developing biomarkers to help validate existing therapeutic targets.

The inaugural AMP AD 1.0 program launched two major activities:

  • The AMP AD Biomarkers in Clinical Trials Project is evaluating the utility of tau imaging for tracking responsiveness to treatment and/or disease by incorporating tau PET into two NIH-funded secondary prevention clinical trials, which include industry support, designed to delay or prevent disease onset. This component includes two anti-amyloid secondary prevention trials which will be completed by 2023.
  • The central goal of the AMP AD Target Discovery and Preclinical Validation Project is to shorten the time between the discovery of potential drug targets and the development of new drugs for Alzheimer’s disease treatment and prevention, by integrating the analyses of large-scale molecular data from human brain samples with network modeling approaches and experimental validation using an open science research model. Over the first 5-year period this component of the AMP AD 1.0 program developed centralized FAIR data infrastructure and delivered a wealth of multi-omic data, many new mechanistic insights and over 500 unique candidate targets. 

The second iteration of this transformative partnership (AMP AD 2.0) was launched in March 2021.

The AMP AD 2.0 program builds on the achievements of the initial Target Discovery component.  Its mission is to enable a precision medicine approach to target and biomarker discovery by expanding the molecular characterization of Alzheimer’s in brain, blood and spinal fluid samples collected in diverse populations.  AMP AD 2.0 is renewing the commitment to open science practices for sharing data, methods and results and continuing the development of the FAIR data infrastructure, the AD Knowledge Portal, and the portal-linked, open-source platform Agora. The AD Knowledge Portal is an informatics platform for rapid sharing of well-annotated, high-quality human multi-omic (genomic, proteomic, metabolomic) data from brain, spinal fluid, and blood samples as well as data from a variety of cell-based and animal models.

Governance

The AD arm of the AMP initiative is managed by an AD steering committee (SC) comprising representatives from NIH, FNIH, the FDA, and participating companies and non-profit and other organizations. The SC operates under the direction of the overall AMP Executive Committee, which includes representatives from NIH, participating industry partners, FDA, and non-profit organizations. The SC meets on a regular basis and is responsible for monitoring ongoing progress towards milestones.

Partners

AMP is a multi-sector partnership managed by the FNIH. AMP partners share expertise and resources to enable the best-informed contributions to science from all participants. AD 1.0 and 2.0 programs have their own set of partners who have signed on to invest in the AMP AD projects. FNIH is the managing partner for both AMP AD programs.

Partners of the AD 1.0 program included the National Institute on Aging (NIA), National Institute of Neurological Disorders and Stroke (NINDS), FDA, AbbVie, Biogen, Eli Lilly, GlaxoSmithKline plc (GSK), Alzheimer’s Association, Alzheimer’s Drug Discovery Foundation, Geoffrey Beene Foundation, Sage Bionetworks, and USAgainstAlzheimer's.

NIA will lead research efforts for AMP AD 2.0. Private funding partners include Eisai Inc., Gates Ventures, and Takeda Pharmaceutical Company Limited. The Alzheimer’s Association and GSK, who have been partners since the beginning of the AMP Alzheimer’s program, will again participate and continue to provide support towards the program’s goals.

Budget 

Disease area Total NIH funding ($M) Total Industry funding ($M) Total non-profit and other organziations funding ($M) Total project funding ($M)
Alzheimer’s Disease 1.0 162 22.2
(+40 in kind)
1.0 185.2
(+40 in kind)
Alzheimer’s Disease 2.0 61.4 8.07 5.38 $74.85

This page last reviewed on March 2, 2021