Treatment with arimoclomol, a conducer of heat shock proteins, delays disease progression of ALS in mice

Source: Kieran, D., Kalmar, B., Dick, J., Riddoch- Contreras, J., Burnstock, G., & Greensmith, L., (2004). Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression of ALS in mice. Nature Medicine, 10(4), 402-405. DOI: 10.1038/nm1021

Summary: Amyotrophic lateral sclerosis (ALS) is a condition where the neurons in the nervous system gradually die eventually resulting in loss of motor function and paralysis. There is currently no cure for the disease, but it has been found that approximately 20% of ALS cases are due to a mutation in the SOD1 gene (in normal function, this gene produces an enzyme that is activated when attached to zinc or copper and serves to break down potentially dangerous oxygen molecules in the body). The mutation of SOD1 causes it to aggregate and no longer function properly, so the goal of the paper is to find a way to delay or prevent the aggregation of SOD1. The study tested the use of arimoclomol which induces heat shock proteins (HSP) to create a heat shock response. The use of arimoclomol increases the availability of HSP proteins which act as chaperone proteins (proteins that protect and maintain the shape of other proteins) delaying the aggregation and loss of shape of the SOD1 protein, delaying the progression of ALS. This treatment results in a 22% increase in the lifespan of those afflicted with ALS.