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Drug Repurposing: Early clinical development (Milestone 7.D)

In Progress

Timeline Start - End

2018 - 2024

Research Implementation Area

Drug Repurposing and Combination Therapy Development

Initiate early clinical development for at least 6 approved drugs or drug combinations for the treatment or prevention of AD and AD-related dementias.

Success Criteria

  • Completion of at least 4 phase II trials with repurposed drugs and/or drug combinations. Successful trials will provide conclusive evidence of therapeutic mechanism/target engagement.

Summary of Key Accomplishments

Fourteen early-stage clinical trials (Phase 1 and 2) testing 11 different FDA-approved drugs repurposed for the treatment of AD/ADRD have been supported. The pilot trials for candesartan (an antihypertensive), nicotine (used for smoking cessation) and metformin (a drug used to treat diabetes) showed positive results, and these repurposed drug candidates have been advanced to larger Phase 2 trials (candesartan and nicotine) and Phase 2/3 trials (metformin). In addition, six early-stage clinical trials have been supported to test six different dietary supplements as potential AD treatments. One of these trials testing the supplement benfotiamine, a synthetic derivative of vitamin B1, showed positive results and can be advanced to Phase 3 testing.

The key accomplishments summary is current as of July 2022. 

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