Efficacy and Safety of Edaravone in Amyotrophic Lateral Sclerosis Patients in Indian Population

Abstract

Amyotrophic lateral sclerosis (ALS), is a progressive fatal neurodegenerative disease. It leads to scarring or hardening of Motor neurons. The cause of ALS remains unknown. Oxidative stress caused by free radicals might be an essential factor in the progression of the disease. Edaravone, is a free-radical scavenger, it has been shown to inhibit motor neuron death in animal models by reducing oxidative stress & it has shown efficacy in a small subset of people with ALS. This study was planned to see the efficacy and safety of Edaravone in Indian population.

Material: This study was a single centric observational study, on use of Edaravone in ALS patients. Who were more than 18 years of age and diagnosed to have possible, probable or definite ALS as per the El Escorial Criteria 2014. Total 30 patients were included. All patients had their Revised ALSFRS-R recorded & SFEMG was done at the time of diagnosis then after 6 months of completion of treatment protocol. They were given Edaravone as per as per defined treatment protocol. The treatment protocol consists of 24 weeks (6 cycles). In cycle 1, the study drug was administered for 14 consecutive days followed by a 2 week drug-free period. In cycle 2 and thereafter, the study drug was administered for first 10 days, followed by 18 days drug-free period. The primary efficacy endpoint was a difference in ALSFRS-R score of at least 20% from base line. Secondary endpoints were change in increase in jitter by 10%. Safety endpoints was include the incidence of adverse drug reactions.

Observation: Total of 30 patients were included in the study and 23 patients completed the treatment protocol. 93.3% of patients reported with weakness of limbs while 80% suffered from atrophy of limbs. 96.7% of patients was having fasciculation.2 patients (6.6%) of subjects receiving Edaravone therapy reported with adverse side-effects.After completing the treatment protocol in the study group. On comparing the mean values of ALSFRS-R score at different end-points, no statistical significance was obtained.

Conclusion: This study failed to demonstrate efficacy of Edaravone to delay the progression of ALS. While the primary desired endpoint was not achieved but there was small improvement in SF-EMG jitter difference of the patients that was not significant statistically. We consider that the study with large sample size results can be helpful to identify the patient population in which Edaravone could be expected to show efficacy.