GNS561 induces lysosomal dysregulation leading to inhibition of autophagy.1 It displayed efficacy against intrahepatic cholangiocarcinoma1 and hepatocellular carcinoma2. GNS561 acted as an anti-fibrotic agent against liver fibrosis in a rat model.3 GNS561-induced cell death is partially caused by inhibition of palmitoyl-protein thioesterase 1 (PPT1) leading to lysosomal dysfunction.4 GNS561 treatment of prostate and ovarian cancer models enhanced STING expression and activation via PPT1 inhibition leading to infiltration of cytotoxic T cells.5 GNS561 represents a potentially novel immunotherapy for cold tumors.
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