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Updated: Feb 15, 2026
Evaluation of Motor Impairment in C. elegans Models of Amyotrophic Lateral Sclerosis
Published on: September 2, 2021
Drugs in clinical development for the treatment of amyotrophic lateral sclerosis
Ana Martinez1, Maria Del Valle Palomo Ruiz1, Daniel I Perez1
1a IPSBB Unit , Centro de Investigaciones Biologicas-CSIC , Madrid , Spain.
Introduction:
Amyotrophic Lateral Sclerosis (ALS) is a fatal motor neuron progressive disorder for which no treatment exists to date. However, there are other investigational drugs and therapies currently under clinical development may offer hope in the near future. Areas covered: We have reviewed all the ALS ongoing clinical trials (until November 2016) and collected in Clinicaltrials.gov or EudraCT. We have described them in a comprehensive way and have grouped them in the following sections: biomarkers, biological therapies, cell therapy, drug repurposing and new drugs. Expert opinion: Despite multiple obstacles that explain the absence of effective drugs for the treatment of ALS, joint efforts among patient's associations, public and private sectors have fueled innovative research in this field, resulting in several compounds that are in the late stages of clinical trials. Drug repositioning is also playing an important role, having achieved the approval of some orphan drug applications, in late phases of clinical development. Endaravone has been recently approved in Japan and is pending in USA.
Insights
Investigational drugs and therapies offer hope for Amyotrophic Lateral Sclerosis (ALS), a progressive motor neuron disease. Several compounds are in late-stage clinical trials, with drug repositioning showing promise for new ALS treatments.
Area of Science:
- Neurology
- Clinical Trials Research
Background:
- Amyotrophic Lateral Sclerosis (ALS) is a progressive, fatal motor neuron disorder with no current cure.
- Despite challenges, research is advancing with new therapies and drug repositioning strategies.
Purpose of the Study:
- To comprehensively review ongoing clinical trials for Amyotrophic Lateral Sclerosis (ALS) up to November 2016.
- To categorize and describe these trials, including biomarkers, biological therapies, cell therapy, and new drug development.
Main Methods:
- Systematic review of ALS clinical trials registered in ClinicalTrials.gov and EudraCT.
- Categorization of trials into key therapeutic areas: biomarkers, biological therapies, cell therapy, drug repurposing, and new drugs.
Main Results:
- Multiple investigational drugs and therapies are in late-stage clinical development for ALS.
- Drug repositioning is a significant strategy, with some applications achieving orphan drug status.
- Recent approval of Endaravone in Japan and pending approval in the USA highlights progress.
Conclusions:
- Despite obstacles, collaborative efforts are driving innovative ALS research and drug development.
- Several promising compounds are nearing the end of clinical trials.
- Drug repositioning strategies are proving effective in advancing ALS treatment options.
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