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Drug Repurposing: Translational bioinformatics and network pharmacology (Milestone 7.B)


Timeline Start - End

2016 - 2022

Research Implementation Area

Drug Repurposing and Combination Therapy Development

Initiate research programs for translational bioinformatics and network pharmacology to support rational drug repositioning and combination therapy from discovery through clinical development.

Success Criteria

Identification of at least 6 existing drugs suitable for repurposing and/or combination therapy for AD and ADRD prevention or treatment.  The drugs selected for repurposing or combination therapy will be prioritized based on:

  • evidence that they modulate disease relevant pathways/networks gained from computational and empirical approaches.

  • preclinical proof-of-efficacy in a relevant model system.

  • availability of biomarkers to monitor target engagement in humans.

  • sufficient evidence of safety for the intended target population.

Summary of Key Accomplishments

Novel uses for existing and failed drugs can save time and cost in bringing new therapeutics to individuals. Investigators funded through NIA’s Drug Repurposing and Combination Therapy Development program, have identified over 20 FDA approved drugs with a potential to be repurposed in AD. An example is bumetanide, an FDA approved drug for the treatment of hypertension and edema; based on computational predictions that utilized publicly available genomic and EHR data and experimental work in animal models, this repurposing candidate has the potential to be effective in individuals with AD that carry the ApoE4 risk factor gene.

NIA’s Alzheimer’s Clinical Trials consortium and clinical trials funding initiatives provide a path for the most promising drug repurposing candidates to be evaluated in human trials for AD/ADRD treatment and prevention. NIA’s AD/ADRD clinical trials program has supported the testing of 10 repurposed drug candidates to date.

The key accomplishments summary is current as of March 2022. 

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