Drug repurposing for Alzheimer's disease and other neurodegenerative disorders

Jeffrey L Cummings; Yadi Zhou; Alexandra Van Stone; Davis Cammann; Reina Tonegawa-Kuji; Jorge Fonseca; Feixiong Cheng

doi:10.1038/s41467-025-56690-4

Drug repurposing for Alzheimer's disease and other neurodegenerative disorders

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

Authors

Jeffrey L Cummings¹, Yadi Zhou², Alexandra Van Stone³, Davis Cammann⁴, Reina Tonegawa-Kuji², Jorge Fonseca⁵, Feixiong Cheng^{2

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Affiliations

¹ Chambers-Grundy Center for Transformative Neuroscience, Department of Brain Health, Kirk Kerkorian School of Medicine, University of Nevada, Las Vegas (UNLV), Las Vegas, NV, 89106, USA. jcummings@cnsinnovations.com.
² Genomic Medicine Institute, Lerner Research Institute, Cleveland Clinic, Cleveland, Ohio, 44195, USA.
³ Roseman University of Health Sciences, Las Vegas, NV, 89135, USA.
⁴ Nevada Institute of Personalized Medicine, University of Nevada Las Vegas (UNLV), Las Vegas, NV, USA.
⁵ Howard R Hughes College of Engineering, Department of Computer Science, University of Nevada, Las Vegas (UNLV), Las Vegas, NV, 89154, USA.
⁶ Department of Molecular Medicine, Cleveland Clinic Lerner College of Medicine, Case Western Reserve University, Cleveland, OH, 44106, USA.
⁷ Case Comprehensive Cancer Center, Case Western Reserve University School of Medicine, Cleveland, Ohio, 44106, USA.
⁸ Cleveland Clinic Genome Center, Lerner Research Institute, Cleveland Clinic, Cleveland, Ohio, 44195, USA.

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.

Publication types

Review

MeSH terms

Alzheimer Disease* / drug therapy
Drug Development
Drug Repositioning* / methods
Humans
Neurodegenerative Diseases* / drug therapy

Abstract

Publication types

MeSH terms

Grants and funding