2025 American Academy of Neurology Abstract Website

Yusra Arafeh¹, Omar Khaled Abdelsalam², Fatma Alzhraa S.A.Osman³, Nereen A. Almosilhy⁴, Sadil Mohammad Bani Khaled⁵, Alamin Awadallah Mohamed Magzoub⁶, Yehia Nabil⁷, Asmaa Zakria⁸
¹Faculty of Medicine, Jordan University of Science and Technology, ²Faculty of Medicine New Mansoura university, New Mansoura, Egypt, ³Faculty of Medicine Minia university, Minia Egypt, ⁴Department of Pharmacology and Toxicology, Faculty of Pharmacy, Tanta University, EGYPT, ⁵Faculty of Medicine,The university of JordanAmman,Jordan, ⁶Faculty of Medicine university of science and technology, sudan, ⁷8.Faculty of Medicine, Zagazig University, Egypt, ⁸Faculty of Medicine, Al-Azhar University, Gaza, Palestine.

Objective:

The study aims to evaluate the effect of arimoclomol on functional status, as measured by the ALSFRS-R scale, and to assess the incidence of adverse events (AEs)

Background:

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.

Design/Methods:

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.

Results:

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.

Conclusions:

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 .