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Drug repurposing for Alzheimer’s disease and other neurodegenerative disorders

Jeffrey L. Cummings, Yadi Zhou, Alexandra Stone, Davis Cammann, Reina Tonegawa‐Kuji, Jorge Ramón Fonseca Cacho, Feixiong Cheng

2025Nature Communications46 citationsDOIOpen Access PDF

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. Repurposed drugs provide a rich source of potential therapies for Alzheimer’s disease (AD) and other neurodegenerative disorders (NDD). In this review, the authors discuss the advantages and challenges of repurposing, offer strategies for overcoming the obstacles and describe the key contributions of repurposing to drug development.

Topics & Concepts

Drug repositioningRepurposingDiseaseAlzheimer's diseaseDrugDrug discoveryMedicinePharmacologyBioinformaticsBiologyInternal medicineEcologyAlzheimer's disease research and treatmentsCholinesterase and Neurodegenerative DiseasesComputational Drug Discovery Methods