Current and emerging treatments for amyotrophic lateral sclerosis

Area of Science:

• Neuroscience
• Pharmacology
• Clinical Trials

Background:

• Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease affecting motor neurons.
• Current ALS management is primarily symptomatic, with riluzole being the sole FDA-approved treatment.

Purpose of the Study:

• To review emerging disease-modifying treatments for ALS.
• To identify compounds investigated for ALS therapy between 1986 and 2009.

Main Methods:

• Comprehensive literature search of Medline database.
• Selection of studies focused on disease-modifying therapies for ALS.
• Review of 48 identified compounds.

Main Results:

• Riluzole demonstrated a modest increase in survival for ALS patients.
• Numerous compounds effective in ALS animal models failed to show significant benefits in human trials.
• Forty-eight compounds were identified and reviewed.

Conclusions:

• Riluzole remains the only drug with a proven, albeit modest, beneficial effect on ALS patient survival.
• Challenges in translating animal model efficacy to human clinical trials persist.
• Methodological issues in animal screening, pharmacokinetic assessment, and clinical trial design may explain negative trial outcomes.