Recent years have seen an explosion of research into increasingly prevalent 
neurodegenerative diseases. Arimoclomol (BRX-220), being developed by CytRx 
Corp, is an oral therapeutic candidate for the treatment of amyotrophic lateral 
sclerosis (ALS), the most common form of motor neuron disease. ALS is a fatal, 
incurable disorder, which can present as sporadic (90 to 95% of cases) or 
familial (5 to 10% of cases) forms. The etiology of sporadic ALS remains unknown 
and much of the understanding of ALS pathogenesis has been derived through study 
of its familial forms; in particular, through study of autosomal dominant 
mutations in the SOD1 (copper/zinc superoxide dismutase) gene, which cause 
approximately 20% of familial ALS cases. Under conditions of excessive stress, 
arimoclomol induces amplification of the cytoprotective heat shock response in 
order to protect motor neurons from death. Comprehensive in vivo and in vitro 
studies demonstrated its effect in the prevention of neuronal loss and promotion 
of motor neuron survival, even after symptom onset. Clinical trials have 
reported good tolerability and safety. This paper discusses the rationale for 
arimoclomol use in ALS, the preclinical and clinical evidence collected to date, 
the likelihood of its promising preclinical results translating to humans, and 
the relevance of this research for neurodegeneration as a whole.