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Alzheimer’s disease and Alzheimer’s disease-related dementias (AD/ADRD) research has advanced at a remarkable pace over the past decade. NIA as well as broader NIH investments in basic to clinical AD/ADRD studies have contributed to the development of disease-modifying drugs; improved diagnostic tools; enhanced understanding of behavioral, lifestyle and genetic factors; and supported critical dementia care and caregiving science.
The NIH-funded clinical trials portfolio is now more diverse than ever in the biological mechanisms and stages of illness being targeted for intervention. Read on for insights into the wide range of interventions and approaches under study.
Increasing the number and diversity of active clinical trials for dementia
With an eye toward precision medicine, NIH is committed to identifying and testing diverse approaches to dementia prevention and treatment. For perspective on how this portfolio has evolved, consider the trends in total NIH-funded AD/ADRD trials, including the proportion that targeted amyloid, over the past 10 years. In 2013, NIH was funding 14 pharmacological trials to prevent or treat AD/ADRD, including five trials (36%) targeting amyloid. In 2023, the number of pharmacological trials grew to 54 with just eight (15%) focused on amyloid.
Current NIH-funded trials focus on multiple disease mechanisms beyond amyloid, including inflammation, oxidative stress, vascular factors, protein folding, and metabolism and bioenergetics. Additionally, we are funding trials targeting the important role of behavioral and lifestyle interventions to improve nutrition, exercise, cognitive function, sleep, and other factors associated with significant risk of dementia disease development and progression.
Expanding the drug development pipeline and data sharing
Our translational funding initiatives — as one of many examples, the Alzheimer’s Disease Drug-Development Program — are instrumental in driving a more diverse therapeutic pipeline. To date, we have supported development of 22 drug candidates that have transitioned to clinical trials. Therapeutic candidates emerging from these initiatives include innovative approaches like immunological, genetic, and stem cell therapies.
All this research progress has also generated a vast amount of data, making data sharing more important than ever. We work to rapidly share data, methods, and results from basic research to clinical trials such as the Anti-Amyloid Treatment in Asymptomatic Alzheimer’s (A4) study . Today, more than 950 unique candidate targets discovered by the Accelerated Medicines Partnership® for Alzheimer's Disease (AMP AD) and other programs are available to all researchers for further exploration through the AD Knowledge Portal and related resources .
Intriguing possibilities in drug repurposing
This robust portfolio is already revealing multiple promising areas of investigation and approaches for preventing or treating dementia. One intriguing strategy is drug repurposing, which is testing drugs that have already been deemed safe and effective by the Food and Drug Administration for treating other conditions for their ability to work against AD/ADRD.
Drug repurposing holds promise to get effective treatments to people living with dementia faster, cheaper, and with fewer safety concerns than development of new and experimental drugs. We currently have multiple studies testing repurposing of common drugs, such as:
- Metformin (approved for diabetes prevention)
- Statins (cholesterol)
- Nilotinib (cancer)
- Siponimod (multiple sclerosis)
Explore the latest targets and strategies, share your input
We encourage the scientific community, the public, and other interested parties to learn more about how dementia research is growing beyond the targets and techniques of the past by exploring our list of Active Alzheimer’s and Related Dementias Clinical Trials and Studies .
Alzheimer’s Disease and Related Dementias Clinical Research Data Sharing Strategic Directions 2025-02-26T00:00:00Z