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Related Experiment Video

Updated: May 8, 2026

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

Published on: July 29, 2007

Drug repurposing in amyotrophic lateral sclerosis (ALS).

Emily Carroll^1,2, Jakub Scaber^1,2, Kilian V M Huber^3,4

¹Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.

Expert Opinion on Drug Discovery

|March 3, 2025

Summary

Drug repurposing offers a promising strategy to find new amyotrophic lateral sclerosis (ALS) treatments by re-evaluating existing drugs. This approach can overcome challenges in traditional drug discovery for ALS, improving patient options.

Keywords:

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

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Last Updated: May 8, 2026

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Published on: July 13, 2015

Area of Science:

Neuroscience
Pharmacology
Drug Discovery

Background:

Drug discovery for amyotrophic lateral sclerosis (ALS) faces significant challenges, with traditional methods yielding limited success.
Repurposing existing, clinically approved drugs for new indications presents a viable alternative strategy.

Purpose of the Study:

To review the challenges in ALS drug discovery.
To examine the potential of drug repurposing for identifying novel ALS treatments.
To outline principles for preclinical and clinical research to bridge the translational gap.

Main Methods:

Literature review of original publications, press releases, and clinical trials.
Discussion of screening approaches in experimental models and computational methods.
Consideration of general principles for preclinical and clinical research.

Main Results:

Drug repurposing offers a strategic advantage over de novo drug discovery for ALS.
A range of screening and computational approaches can be employed.
Bridging the translational gap requires careful preclinical and clinical research design.

Conclusions:

Drug repurposing holds significant potential to enhance therapeutic options for ALS patients.
Stringent preclinical research is essential to identify promising compounds.
Collaborative efforts across academia, industry, and stakeholders are crucial for successful clinical translation of repurposed therapies.