Related Experiment Video
Updated: Apr 29, 2026
15:48
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
Published on: July 29, 2007
9.0K
Innovative therapies under clinical development for ALS treatment: small molecules.
Loreto Martinez-Gonzalez1,2, Cecilia Sanchez-Santos1,2, Chi-Chen Sarah Huang3,4
1Biomedicine Department, Centro de Investigaciones Biologicas "Margarita Salas"-CSIC, Madrid, Spain.
Expert Opinion on Investigational Drugs
|April 28, 2026
Summary
This review details the evolving Amyotrophic Lateral Sclerosis (ALS) clinical trial landscape, focusing on small-molecule therapeutics progressing through research. It highlights advancements in trial platforms and precision medicine for ALS treatment development.
Area of Science:
- Neurology
- Clinical Pharmacology
- Biomedical Research
Background:
- The Amyotrophic Lateral Sclerosis (ALS) clinical trial landscape is dynamic, facing challenges but seeing increased novel therapeutics.
- Previous work in 2022 provides a foundation for this updated review of the ALS pipeline.
Purpose of the Study:
- To provide a comprehensive overview of the current state of small-molecule drug development for ALS.
- To focus on agents that have entered or progressed through clinical evaluation from 2022 to the end of 2025.
Main Methods:
- Systematic review of ALS clinical trials registered in the United States (ClinicalTrials.gov) and European Union (EU Clinical Trials Register/CTIS).
Main Results:
- The ALS therapeutic pipeline is expanding with diverse biological targets and approaches.
- Advanced clinical trial platforms and adaptive strategies are increasing the breadth of explored therapies.
- Precision medicine, utilizing Artificial Intelligence (AI), is enhancing patient selection and stratification for ALS trials.
Conclusions:
- The ALS clinical trial landscape is rapidly evolving with novel small-molecule therapeutics.
- Advanced trial designs and AI-driven precision medicine offer promising avenues to overcome challenges in treating this heterogeneous disease.
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