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Updated: Jul 13, 2026

Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis (ALS)
Published on: March 17, 2012
Current clinical trials in amyotrophic lateral sclerosis
Jaydeep M Bhatt1, Paul H Gordon
1Columbia University, Eleanor and Lou Gehrig MDA/ALS Research Center, Department of Neurology, Neurological Institute, New York, NY 10032, USA.
Amyotrophic lateral sclerosis (ALS) lacks effective treatments beyond riluzole. This review examines ongoing clinical trials and emerging drug candidates for ALS, focusing on scientific evidence and trial design.
Area of Science:
- Neuroscience
- Neurology
- Drug Development
Background:
- Amyotrophic lateral sclerosis (ALS) involves motor neuron degeneration, with limited therapeutic options available.
- Riluzole, approved a decade ago, remains a primary treatment, highlighting the urgent need for novel therapies.
- Advances in drug discovery are expanding the pipeline of potential ALS treatments.
Purpose of the Study:
- To review clinical trial methodologies for amyotrophic lateral sclerosis.
- To systematically evaluate drugs currently in Phase I, II, and III clinical trials for ALS.
- To emphasize the scientific rationale and trial design for each investigational drug.
Main Methods:
- Literature review of clinical trial methodology in ALS.
- Systematic evaluation of drugs in active clinical development (Phase I-III).
- Analysis of scientific evidence supporting drug selection and trial design.
Main Results:
- Identification of numerous potential therapeutic agents for ALS in various trial phases.
- Assessment of the scientific basis for drug candidates.
- Evaluation of the strengths and weaknesses of current ALS clinical trial designs.
Conclusions:
- The landscape of ALS drug development is rapidly evolving with promising new agents.
- Rigorous clinical trial design and strong scientific evidence are crucial for advancing ALS therapies.
- Continued research and development are essential to address the unmet needs in ALS treatment.
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