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Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis ALS
Published on: March 17, 2012
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Clinical studies in amyotrophic lateral sclerosis
Johannes Dorst1, Angela Genge2
1Department of Neurology, University of Ulm, Ulm, Germany.
Current Opinion in Neurology
|August 9, 2022
Summary
Recent amyotrophic lateral sclerosis (ALS) clinical studies show promise for treatments like edaravone and tofersen. New study designs incorporate biomarkers and progression rates for better therapeutic evaluation.
Area of Science:
- Neurology
- Clinical Trials
- Drug Development
Background:
- Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease with limited treatment options.
- Recent clinical research has explored novel therapeutic agents and improved study methodologies.
Purpose of the Study:
- To review significant recent clinical studies in ALS.
- To analyze their impact on clinical practice, methodology, and future research directions.
- To highlight promising therapeutic agents and advancements in clinical trial design.
Main Methods:
- Focus on studies with positive primary endpoints or post hoc analyses.
- Inclusion of drugs such as edaravone, sodium phenylbutyrate-taurursodiol, rasagiline, and tofersen.
- Examination of recent developments in clinical trial design, including inclusion criteria, stratification, and outcome parameters.
Main Results:
- Edaravone approved in the US, with oral formulations in development.
- Ongoing follow-up studies for sodium phenylbutyrate-taurursodiol; planned for rasagiline and high-caloric nutrition.
- Tofersen Phase III study yielded promising, though negative, results.
- Neurofilament light chain (NfL) levels recognized as a standard outcome parameter.
- Consideration of progression rate for therapeutic response and stratification.
Conclusions:
- Several substances show potential for ALS treatment.
- Advancements in clinical trial design, including NfL and progression rate, are crucial for future research.
- Continued investigation and optimized study methodologies are essential for developing effective ALS therapies.
