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Expert opinion on drug discovery
Apr, 2025;20 (4): 447-464.

Drug repurposing in amyotrophic lateral sclerosis (ALS).

Emily Carroll, Jakub Scaber, Kilian V M Huber, Paul E Brennan, Alexander G Thompson, Martin R Turner, Kevin Talbot
Author Information
  1. Emily Carroll: Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
  2. Jakub Scaber: Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
  3. Kilian V M Huber: Centre for Medicines Discovery, Nuffield Department of Medicine, University of Oxford, Oxford, UK. ORCID
  4. Paul E Brennan: Centre for Medicines Discovery, Nuffield Department of Medicine, University of Oxford, Oxford, UK.
  5. Alexander G Thompson: Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
  6. Martin R Turner: Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
  7. Kevin Talbot: Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
PMID: 40029669 DOI: 10.1080/17460441.2025.2474661

Abstract

INTRODUCTION: Identifying treatments that can alter the natural history of amyotrophic lateral sclerosis (ALS) is challenging. For years, drug discovery in ALS has relied upon traditional approaches with limited success. Drug repurposing, where clinically approved drugs are reevaluated for other indications, offers an alternative strategy that overcomes some of the challenges associated with de novo drug discovery.
AREAS COVERED: In this review, the authors discuss the challenge of drug discovery in ALS and examine the potential of drug repurposing for the identification of new effective treatments. The authors consider a range of approaches, from screening in experimental models to computational approaches, and outline some general principles for preclinical and clinical research to help bridge the translational gap. Literature was reviewed from original publications, press releases and clinical trials.
EXPERT OPINION: Despite remaining challenges, drug repurposing offers the opportunity to improve therapeutic options for ALS patients. Nevertheless, stringent preclinical research will be necessary to identify the most promising compounds together with innovative experimental medicine studies to bridge the translational gap. The authors further highlight the importance of combining expertise across academia, industry and wider stakeholders, which will be key in the successful delivery of repurposed therapies to the clinic.

Keywords

Amyotrophic lateral sclerosis (ALS) drug discovery drug repurposing experimental medicine neurodegeneration

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MeSH Term

Drug Repositioning
Humans
Amyotrophic Lateral Sclerosis
Animals
Drug Discovery
Translational Research, Biomedical
Journal Article Review
Article has an altmetric score of 12

Word Cloud

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