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Review
. 2025 Feb 19;16(1):1755.
doi: 10.1038/s41467-025-56690-4.

Drug repurposing for Alzheimer's disease and other neurodegenerative disorders

Jeffrey L Cummings  1 , Yadi Zhou  2 , Alexandra Van Stone  3 , Davis Cammann  4 , Reina Tonegawa-Kuji  2 , Jorge Fonseca  5 , Feixiong Cheng  2   6   7   8
Affiliations
Review

Drug repurposing for Alzheimer's disease and other neurodegenerative disorders

Jeffrey L Cummings et al. Nat Commun. .

Abstract

Repurposed drugs provide a rich source of potential therapies for Alzheimer's disease (AD) and other neurodegenerative disorders (NDD). Repurposed drugs have information from non-clinical studies, phase 1 dosing, and safety and tolerability data collected with the original indication. Computational approaches, "omic" studies, drug databases, and electronic medical records help identify candidate therapies. Generic repurposed agents lack intellectual property protection and are rarely advanced to late-stage trials for AD/NDD. In this review we define repurposing, describe the advantages and challenges of repurposing, offer strategies for overcoming the obstacles, and describe the key contributions of repurposing to the drug development ecosystem.

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Conflict of interest statement

Competing interests: J.C. has provided consultation to Acadia, Acumen, ALZpath, Axsome Artery, Biogen, Biohaven, Bristol-Myers Squib, Eisai, Fosun, GAP Foundation, Janssen, Karuna, Kinoxis, Lighthouse, Lilly, Lundbeck, LSP/eqt, Merck, MoCA Cognition, New Amsterdam, Novo Nordisk, Optoceutics, Otsuka, Oxford Brain Diagnostics, Praxis, Prothena, ReMYND, Roche, Scottish Brain Sciences, Signant Health, Simcere, sinaptica, TrueBinding, and Vaxxinity pharmaceutical, assessment, and investment companies. J.C. is the owner of the Neuropsychiatric Inventory. Y.Z., A.S., D.C., R.T.-K., J.F., and F.C. declare no competing interests.

Figures

Fig. 1
Fig. 1. The clinical development pathway for a repurposed agent and the alternatives pursued if needed, to characterize the repurposed agent for the new indication.
After entering at Phase 2 or Phase 3, reverse translation may be needed to resolve dosing or safety issues in Phase 1 studies or mechanistic questions in non-clinical studies (copyright J Cummings; Illustrator M de la Flor, phD).
See this image and copyright information in PMC

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