Advanced Treatment of Amyotrophic Lateral Sclerosis (ALS)

Abstract

Background: Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neuromuscular disease that affects approximately 5 of every 100,000 individuals, Approximately half of ALS patients die within 30 mo of symptom onset and the majority do not survive beyond 5 years, Currently, riluzole and edaravone are the only United States Food and Drug Administration (FDA)–approved treatment options Methods: Outcome (the change in ALS Functional Rating Scale–Revised, ALSFRSR, from baseline) was projected for placebo patients through 48 weeks and compared with 48-week edaravone or 24-week edaravone after switching from placebo. Results: A total of 123 patients received open-label treatment (65 edaravoneedaravone; 58 placebo-edaravone). The projected ALSFRS-R decline for placebo from baseline through week 48 was greater than for 48-week edaravone (P < .0001). For patients switching from placebo to edaravone, ALSFRS-R slope approached that of continued edaravone for 48 weeks. ALSFRS-R decline did not differ between actual and projected edaravone through week 48. Conclusions: Compared with placebo, these analyses suggest that edaravone is beneficial in ALS patients even after 6 mo of receiving placebo, and efficacy is maintained for up to 1 year.