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SC-2001 is a small-molecule obatoclax analog and STAT3 inhibitor developed by researchers at National Taiwan University Hospital, National Yang-Ming University, Taipei Veterans General Hospital, and the Industrial Technology Research Institute. It acts by enhancing the expression and activity of the protein tyrosine phosphatase SHP-1 (Src homology region 2 domain-containing phosphatase-1) via the transcription factor RFX-1. This upregulation of SHP-1 leads to the dephosphorylation and inactivation of STAT3 (specifically at Tyr 705), subsequently downregulating STAT3-regulated proteins such as Mcl-1, survivin, and cyclin D1. Additionally, SC-2001 directly inhibits the protein-protein interactions between Mcl-1 and Bak. It has demonstrated preclinical efficacy in inducing apoptosis and inhibiting tumor growth in breast cancer and hepatocellular carcinoma (HCC), including overcoming sorafenib resistance in HCC models.
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