Drug repurposing in amyotrophic lateral sclerosis (ALS)

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.