The effect of arimoclomol treatment of fibroblasts on the activity of causal enzymes of Fabry disease and neuronal ceroid lipofuscinosis

We investigated the effect of treatment of fibroblasts with arimoclomol, a heat shock protein co-inducer, on the causal enzymes of Fabry disease and neuronal ceroid lipofuscinosis (NCL). The results revealed that the treatment of female fibroblasts but not male ones with arimoclomol increased the activity of alpha-galactosidase A (αGLA), of which enzyme deficiency is responsible for Fabry disease. The effect of the arimoclomol treatment was not observed in the enzyme of male cells and in the mutated enzyme of Fabry disease patients. The increased activity of normal α-GLA remaining in female patients could be applied to treatment of their symptoms of Fabry disease.