2088 Amyotrophic Lateral Sclerosis Medications: Decoding Efficacy and Safety in the Pursuit of Hope

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INTRODUCTION: Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease characterized by a decline in motor function, often assessed using the ALS Functional Rating Scale-Revised (ALSFRS-R). While multiple pharmacological interventions have been explored, their efficacy in slowing disease progression remains uncertain. This study evaluates the impact of traditional and new medications on functional and respiratory decline in ALS patients. METHODS: A meta-analysis was conducted to evaluate changes in ALSFRS-R scores, FVC, and SVC across different medications compared to matched controls. Linear regression analysis was used to assess the relationship between treatment type and changes in ALSFRS-R scores and vital capacities over time. RESULTS: The experimental group exhibited a decline in ALSFRS-R scores from a mean of 37.8 (SD 3.4) at baseline to 32.2 (SD 5.5) at follow-up, while the control group dropped from 38.8 (SD 2.6) to 30.4 (SD 7.9). Regression analysis revealed a significant effect of treatment on ALSFRS-R scores (β = -1.19, 95% CI: -1.88 to -0.51 for Tofersen; β = -4.39, 95% CI: -8.32 to -0.45 for Edaravone). Mexiletine showed minimal change with a β of -0.65 (95% CI: -0.02 to -1.33). Vital capacities declined, with forced vital capacity (FVC) and slow vital capacity (SVC) showing slower reductions in the experimental groups. Significant intercepts were found across models (all p < 0.05). TUDCA, Acetyl L-Carnitine, and Perampanel notably slowed the decline in ALSFRS-R scores, with reductions at follow-up of 1.5, 6.7, and 14.9 points. in terms of respiratory outcomes, Tirasemtiv showed a significant decline in slow vital capacity of 10.34 points, while RELYVRIO (AMX0035) significantly slowed the respiratory decline (2.294×10-16). CONCLUSIONS: All ALS patients experienced functional decline, but medication efficacy varied. Experimental treatments showed potential in slowing ALS progression, particularly in ALSFRS-R score preservation and respiratory function maintenance. These findings underscore the need for continued exploration of novel therapeutic options to optimize ALS management and improve patient outcomes.

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