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Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice
Published on: February 24, 2026
Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).
R G Miller1, J D Mitchell, M Lyon
1California Pacific Medical Center, Department of Neurosciences, 2324 Sacramento Street, Suite 150, San Francisco, California 94115, USA. millerrx@sutterhealth.org
Riluzole (riluzole) at 100 mg daily may extend survival for amyotrophic lateral sclerosis (ALS) patients by approximately two to three months. This treatment is generally safe, offering a modest survival benefit for ALS management.
Area of Science:
- Neurology
- Pharmacology
Background:
- Riluzole is approved for amyotrophic lateral sclerosis (ALS) treatment globally.
- Its clinical utility is debated due to cost and modest efficacy.
Purpose of the Study:
- To evaluate riluzole's efficacy in prolonging survival in ALS patients.
- To assess its impact on delaying tracheostomy and mechanical ventilation.
Main Methods:
- Systematic review of randomized controlled trials (RCTs) identified through comprehensive database searches (Cochrane, MEDLINE, EMBASE).
- Included adults diagnosed with ALS, comparing riluzole treatment against placebo.
- Primary outcome: pooled hazard ratio for tracheostomy-free survival; Secondary outcomes: mortality, neurological function, muscle strength, and adverse events.
Main Results:
- Four RCTs with 974 riluzole and 503 placebo patients were analyzed.
- Riluzole 100 mg/day showed a statistically significant survival benefit in a homogeneous subgroup (P=0.042, HR=0.80).
- Overall analysis, including heterogeneous trials, approached significance (P=0.056, HR=0.84), indicating a 9% increase in one-year survival probability. Mild benefits in bulbar/limb function noted, but not muscle strength. Increased alanine transferase levels observed.
Conclusions:
- Riluzole 100 mg daily appears safe and likely extends median survival by 2-3 months for ALS patients.
- The drug offers a modest but potentially valuable survival benefit in ALS management.
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