Arimoclomol is an Orally Active Co-Inducer of Heat Shock Proteins (HSP)

HSP (Heat shock proteins) are a group of proteins induced by heat shock, the most prominent members of this group are a class of functionally related proteins involved in the folding and unfolding of other proteins. HSP expression is increased when cells are exposed to elevated temperatures or other stress. In addition, HSP have been classified into six families according to their molecular mass (e.g., Hsp60, Hsp70 and Hsp90). Cells respond to heat shock or other stresses with the rapid synthesis of HSP. With the development of stress conditions, many cellular proteins will be partially or completely degenerated or deformed. Moreover, HSP recognizes this, binds to damaged proteins, and stabilizes and refolds them, thereby preventing or dissolving other irreversible aggregations.

Arimoclomol (also known as BRX-220) is an orally active heat shock proteins (HSP) co-inducer. Arimoclomol can amplify HSP gene expression and promote the induction of HSP, thereby enhancing the endogenous cytoprotective mechanism under cell stress. In the case of excessive stress, Arimoclomol induces the amplification of the cytoprotective heat shock response to protect motor neurons from death. Importantly, this compound has the potential for amyotrophic lateral sclerosis (ALS) research. For example, Arimoclomol improves survival and muscle function in a mouse model of motor neuron disease. Furthermore, Arimoclomol has a protective effect against CCK-induced acute pancreatitis in rats. Specifically, Arimoclomol treatment significantly increases the pancreatic levels of HSP60 and HSP72 in the animals.

To sum up, Arimoclomol is an orally active co-inducer of HSP,  and has the potential for amyotrophic lateral sclerosis (ALS) research.


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[2] Veena Lanka, et al. Expert Opin Investig Drugs. 2009 Dec;18(12):1907-18.