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Niclosamide is an inhibitor of the Stat3 signaling pathway. Niclosamide inhibits the activation, nuclear translocation and transactivation of Stat3. Niclosamide displays selectivity for Stat3 over Stat1, Stat5, JAK1, JAK2 and Src kinases. Niclosamide inhibits the transcription of Stat3 target genes and induces cell growth inhibition, apoptosis and cell cycle arrest of cancer cells with constitutively active Stat3. Niclosamide is also a FRAP inhibitor via reversible inhibition of mTORC1 signaling and stimulates autophagy in vitro. Niclosamide displays antineoplastic effects in acute myelogenous leukemia (AML) stem cells.
1. Balgi, Aruna D., et al., 2009. Screen for chemical modulators of autophagy reveals novel therapeutic inhibitors of mTORC1 signaling. PloS one. 4(9): e7124. PMID: 19771169
2. Jin, Yanli., et al., 2010. Antineoplastic mechanisms of niclosamide in acute myelogenous leukemia stem cells: inactivation of the NF-kappaB pathway and generation of reactive oxygen species. Cancer research. 70(6): 2516-27. PMID: 20215516
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