Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that shows the death of motor neurons in the brain and spinal cord. Arimoclomol has been investigated for its potential therapeutic role in delaying the progression of ALS by enhancing heat-shock protein activity.

We searched PubMed, Web of Science, Scopus, and the Cochrane Library for RCTs that compared arimoclomol to placebo in patients with ALS. The main outcome was measured by ALSFRS-R scale score for functional status, and the secondary outcome measures were the incidence of  AEs. Additionally, we used the Cochrane Handbook and the PRISMA criteria to perform this systematic review and meta-analysis.

Our study included three RCTs after an initial pool of 198 records. There was no statistical significance in ALSFRS-R scores between the Arimoclomol and control groups, with an MD of 0.17 (95% CI: [-2.85, 3.19]; p = 0.91). However, sensitivity analysis showed a statistically significant difference between the two groups with MD of  1.90( 95% CI: [0.12, 3.68]; P < 0.04). Furthermore, the incidence of serious adverse events was found to have an OR of 1.11 with a 95% CI ranging from 0.29 to 4.28, which did not show a statistically significant difference.

Arimoclomol showed no significant impact on the progression of ALS and did not give any evidence of an increase in risk for severe adverse events. However, future studies with larger populations, biomarkers, and more sensitive outcome measures are necessary to evaluate its full  potential efficacy in ALS .