Alzheimer’s Disease

Overview

The National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), 10 biopharmaceutical companies, and multiple non-profit organizations launched an unprecedented public-private partnership in February 2014. Managed through the Foundation for the NIH (FNIH), the Accelerating Medicines Partnership (AMP) brings high-level government, industry, and non-profit organization partners together to identify and validate the most promising biological targets for therapeutics. The partners designed a bold milestone-driven research plan to tackle this challenge for Alzheimer’s disease, as well as for type 2 diabetes and the autoimmune disorders of rheumatoid arthritis and systemic lupus erythematosus (lupus). Importantly, the AMP data and analyses are being made publicly available to the broad biomedical community. This fact sheet addresses the AMP research plan for Alzheimer’s disease.

About Alzheimer’s Disease

Dementia, of which Alzheimer’s disease is the most common form, is estimated to affect 36 million people worldwide. This number is expected to rise to 115 million by 2050 unless an effective therapeutic is developed. The financial toll of dementia is already staggering: in the U.S. alone, the costs of caring for people over 70 with dementia were estimated to be as high as $215 billion in 2010. AD 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.

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. This reflects the need for a change in how the academic, biopharmaceutical, and government sectors that participate in Alzheimer’s disease research and therapy development generate, share, and use knowledge to propel development of critically needed therapies. It is becoming apparent that real progress in developing effective therapies will require transforming the Alzheimer’s research and drug development process into one that is participatory, collaborative, well-integrated, and iterative. The AMP-AD program is 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. AMP-AD has two components, the first Biomarkers Project explores 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. The second Target Discovery and Preclinical Validation Project seeks to shorten the time between discovery of potential drug targets to development of new drugs for Alzheimer’s treatment and prevention. The project integrates analysis of large-scale molecular data from human brain samples with network modeling approaches and experimental validation, and enables rapid, broad sharing of data and analytical tools.

AMP-AD Knowledge Portal

Launched in March 2015, the AMP-AD Knowledge Portal is an informatics platform for rapid sharing of well-annotated, multi-omic data (genomic, proteomic, metabolomic) and analytical tools developed by the AMP-AD Target Discovery Consortium and associated open-science consortia.  The AMP-AD Knowledge Portal hosts rich molecular and clinical on more than 2,000 human brains at all stages of Alzheimer’s disease, including a subset of brains from AD-related dementias.  

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 organizations. The SC operates under the direction of the overall AMP Executive Committee (EC), 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.

Budget: 5 years ($185.2 Million Total Project Funding)

Disease area Total NIH funding ($M) Total Industry funding ($M) Total non-profit funding ($M) Total project funding ($M)
Alzheimer’s Disease 162 22.2
(+40 in kind)
1.0 185.2
(+40 in kind)

This page last reviewed on April 24, 2018