Amyotrophic Lateral Sclerosis (ALS) is a progressive and fatal neurodegenerative disorder that primarily affects the motor neurons in the motor cortex, spinal cord, and brainstem. Ongoing research is focused on developing therapeutic strategies to modify the disease’s progression, with Riluzole being the drug currently shown to extend survival. Among emerging therapeutic options, Arimoclomol has demonstrated benefit in preclinical studies. It acts as a co-inducer of heat shock protein-70 (HSP70) and aims to enhance the clearance of misfolded proteins, a hallmark of sporadic ALS pathology.

Three studies encompassing 367 patients were analyzed. Evaluation of the ALSFRS-R scores indicated non-significant improvements in studies with lower drug dosages, whereas no differences were observed in the ORARIALS-01 between treatment groups. In two of these studies, adverse events were generally mild and unrelated to the drug. However, the ORARIALS-01 study observed a higher treatment discontinuation rate attributed to the drug. Reported adverse events included dyspnea, nausea, anorexia, vomiting, duodenal ulcer, muscle weakness, pain, anxiety, depression, headache, gastrostomy tube complications, apnea, and coronary artery disease. 

Current data do not support the use of Arimoclomol for patients with ALS. Although the drug has been demonstrated to be neuroprotective in animal models, this characteristic was not reproducible in patients with ALS and did not significantly change the disease course. Further studies should be conducted to evaluate its possible efficacy in specific groups of patients.