Arimoclomol, an amplifier of heat shock protein expression involved in cellular 
stress response, has emerged as a potential therapeutic candidate in amyotrophic 
lateral sclerosis (ALS) in recent years. Treatment with arimoclomol was reported 
to improve survival and muscle function in a mouse model of motor neuron 
disease. Several single- and multiple-dose safety studies have been completed in 
healthy control subjects. A 3-month Phase IIa study in people with ALS 
demonstrated safety at dosages up to 300 mg/day and another study is currently 
recruiting participants with familial ALS caused by mutations in the superoxide 
dismutase gene. We review the rationale for testing arimoclomol in sporadic and 
familial ALS in the context of available safety and pharmacokinetic data. 
Published and unpublished literature relative to the drug in the past two 
decades is discussed. The current review attempts to bring together our existing 
understanding of the actions of arimoclomol with the disease profile of ALS. The 
pharmacological profile of arimoclomol and the available preclinical data make 
it a promising therapeutic possibility in ALS.