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Updated: Jun 27, 2026
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Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
ALS drug development: reflections from the past and a way forward
Swati Aggarwal1, Merit Cudkowicz
1Department of Neurology, Massachusetts General Hospital, Neurology Clinical Trials Unit, Charlestown, Massachusetts 02129, USA. spaggarwal@partners.org
Summary
Despite advances in understanding amyotrophic lateral sclerosis (ALS) pathogenesis, few treatments show efficacy. This review critically examines past ALS clinical trials, therapy selection, and challenges to improve future drug development.
Area of Science:
- Neurology
- Neuroscience
- Drug Development
Background:
- Amyotrophic lateral sclerosis (ALS) pathogenesis understanding has advanced significantly, leading to numerous drug candidates.
- Over 32 unique compounds have been evaluated in clinical trials for ALS treatment.
- Currently, riluzole remains the sole approved therapy shown to prolong survival in ALS patients.
Purpose of the Study:
- To provide a critical overview of historical amyotrophic lateral sclerosis (ALS) clinical trials.
- To analyze the process of selecting therapies for human testing in ALS.
- To identify challenges in ALS clinical trial design and execution and propose future directions.
Main Methods:
- Review of past amyotrophic lateral sclerosis (ALS) clinical trial data.
- Analysis of drug selection criteria for ALS therapeutic development.
- Examination of methodological challenges in conducting ALS clinical trials.
Main Results:
- A large number of compounds (at least 32) have been tested in ALS clinical trials.
- Riluzole is the only drug currently approved that extends survival.
- Significant challenges persist in ALS clinical trial design and execution.
Conclusions:
- Despite extensive research and numerous drug trials, effective treatments for amyotrophic lateral sclerosis (ALS) remain limited.
- Improvements in clinical trial design, patient selection, and outcome measures are crucial for advancing ALS therapy development.
- Future strategies should focus on overcoming existing challenges to accelerate the discovery of effective ALS treatments.
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