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Through its open science approaches, NIH accelerates the pace and efficiency of research progress. The agency’s investments in centralized data-sharing platforms and other technologies make it possible for scientists to share data, tissue samples, methods, and other crucial research resources more broadly and effectively.
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The resulting large sets of data — often referred to as big data — provide the basis for discoveries that are revealing new molecular mechanisms of Alzheimer’s and related dementias, providing more pathways to potential therapeutic targets and biomarkers.
NIH’s ability to quickly respond to the urgent need for vaccine and treatment development for the coronavirus pandemic was made possible through experience from its already-established open science initiatives such as the Accelerating Medicines Partnership programs for Alzheimer’s and Parkinson’s ; M2OVE-AD, Resilience-AD, and Psych-AD ; MODEL-AD ; and TREAT-AD . These and similar NIH initiatives have transformed the way that scientists collaborate rather than compete, share their data and biological samples , work together to discover new biological mechanisms of disease, and find new drug candidates for testing.
Sharing population data of cognitive decline
NIH-supported behavioral studies also make data freely available to researchers. Decades of data collected from research participants about risk factors, their symptoms, and the development of cognitive impairment and dementia are available for analysis. For example, researchers share data from the NIA-supported Harmonized Cognitive Assessment Protocol , through which investigators are examining cognitive change and dementia in about 3,500 older adults who already participate in the Health and Retirement Study .
In addition, similar studies are sharing data collected from research participants in Chile, China, England, the European Union, India, Mexico, and South Africa.
Developing new animal models of dementia
NIH provides new animal models for basic research or for therapy development. For example, to date, research teams that are part of the NIA-supported Model Organism Development and Evaluation for Late-Onset Alzheimer’s Disease (MODEL-AD) consortium have created more than 50 genetically modified mouse models. These mice are available to the research community through the JAX AD Mouse Model Resource , and the data, protocols, and other resources are available through the Experimental Tools section of the AD Knowledge Portal .
The MODEL-AD consortium also provides resources for rigorous preclinical efficacy testing of drug candidates. Researchers from academia and industry can apply for access to these via the STOP-AD Portal .
In addition, the National Institute of Neurological Disorders and Stroke’s (NINDS) Development and Validation of Advanced Mammalian Models for frontotemporal disorder, vascular dementias, Lewy body dementia, and mixed dementias resulted in several new research projects . These will help fill the critical need for next-generation animal models for many types of dementia.
Expanding the research workforce
NIH is committed to expanding the biomedical research workforce dedicated to Alzheimer’s and related dementias research. As another result of increased federal investments in this area, NIA created many new funding opportunities for scientists and invited NIH-funded investigators from other research areas to apply for supplemental funding relevant to dementias.
Programs are aimed at trainees at multiple points along the career trajectory, from students through early and senior investigators. Examples include:
- Developing clinical trial experts: Finding treatments for Alzheimer’s and related dementias will depend on well-designed and expertly conducted clinical trials. To grow the number of specialized clinical trial experts, NIA supports the Institute on Methods and Protocols for Advancement of Clinical Trials in Alzheimer’s Disease and Related Dementias .
The comprehensive training program is designed to provide education and tools to establish a national cohort of qualified, diverse investigators to continue to enhance and improve Alzheimer’s and related dementias treatment studies. Candidates are selected for their diverse demographic characteristics (such as age, race, and ethnicity), their specialties (such as physicians, psychologists, or statisticians), backgrounds (rural or urban), and career stage. The inaugural class , which finished the course in 2020, included 25 female, 15 nonwhite, and six Hispanic/Latino trainees. Enhancing the diversity of researchers focused on aging and dementia: NIA recently expanded its investment in the Resource Centers for Minority Aging Research NIA now supports eight new resource centers focused on priority areas of social and behavioral science related to Alzheimer’s and related dementias. The overarching centers’ mission is to:
- Enhance the diversity of the aging research workforce by mentoring promising scientists from underrepresented groups for sustained careers in aging research in priority areas of social, behavioral, and economic research on aging
- Develop infrastructure to promote advances in these areas while simultaneously increasing the number of researchers focused on health disparities and the health and well-being of older adults from minority groups
- Encouraging behavioral and social sciences research skills: In March 2021, NIA released a new funding opportunity for Short Courses on Interdisciplinary Behavioral and Social Sciences Research on Alzheimer’s and Related Dementias . These short courses, which are part of the NIH Research Education Program , will support skills development for the Alzheimer’s and related dementias workforce. The funding opportunity will also encourage the recruitment of new and diverse investigators into this area of research and to provide course participants with opportunities to interact with one another and course faculty.
According to an analysis published by NIA in 2020, those new opportunities enabled many new investigators, as well as seasoned scientists from other research areas, to study Alzheimer’s and related dementias.
Program Spotlight: Compressing the timeline for developing treatments
NIH recently announced the next version of the Accelerating Medicines Partnership® for Alzheimer’s Disease (AMP® AD) . During the first version, which began in 2014, the AMP AD program’s open science, big data approach enabled research teams to identify and make more than 500 unique candidate targets for this complex disease publicly available. In the second version, NIA is again leading research efforts that now include funding a data coordinating center at Sage Bionetworks, as well as funding several multi-institutional, cross-disciplinary academic research teams .
Since its launch seven years ago, AMP AD has made possible many new disease insights on the role of the genome, proteome, metabolome, and microbiome, based on data collected from human samples and animal and cell-based models. Recent AMP AD findings , described in the Biomarker Research and Disease Mechanisms sections, showcase research advances related to the discovery of new candidate targets, identification of molecular subtypes, and new potential biomarkers for a precision medicine approach to therapy development.
The AD Knowledge Portal , an informatics data sharing platform that began as the data repository for the AMP AD Target Discovery Program, and the portal-linked, open-source platform Agora have enabled access to a vast amount of high-quality molecular data, analytical results, and candidate targets generated by AMP AD research teams.
The AD Knowledge Portal now includes data and resources from other NIA-supported team-science projects operating under open science principles.
The second version of AMP AD will continue to raise the standards for rigorous and reproducible research, and it will expand the characterization of Alzheimer’s through the analysis of human brain samples. This will enable AMP AD research teams to refine the characterization of new targets, discover new biomarkers, define disease subtypes, and increase the understanding of causative factors and steps in disease progression. The knowledge gained will inform the development of therapies that can be tailored to an individual’s unique disease risk profile.
Private sector funding partners for the second version of AMP AD include Eisai Inc., Gates Ventures, and Takeda Pharmaceutical Company Limited. In addition, the Alzheimer’s Association and GlaxoSmithKline plc (GSK) — AMP AD partners since the launch of the program in 2014 — continue to participate.
Advancing research
The AMP AD program demonstrates how open science can enable the scientific community to investigate difficult scientific questions and jumpstart new drug discovery projects.
More than 3,000 researchers around the world have used the NIH-supported AD Knowledge Portal to advance research on Alzheimer’s and related dementias.
NIH stands against structural racism in biomedical research
Within the biomedical research enterprise, structural and institutional racism has resulted in inequitable access to funding, training, and workforce opportunities, and this limits the pace of scientific progress.
In March 2021, NIH established the UNITE initiative to address structural racism and promote racial equity and inclusion within the NIH-supported and the greater scientific community.
UNITE has five committees with the following specific aims:
U — Understanding stakeholder experiences through listening and learning
N — New research on health disparities, minority health, and health equity
I — Improving the NIH culture and structure for equity, inclusion, and excellence
T — Transparency, communication, and accountability with our internal and external stakeholders
E — Extramural research ecosystem: changing policy, culture, and structure to promote workforce diversity
NIH is committed to instituting new ways to support diversity, equity, and inclusion, and identifying and dismantling any policies and practices that may harm our workforce and our science.