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Uroacitides (CDA-II) is a small molecule drug originally isolated from healthy human urine and developed as an anti-tumor agent. It functions primarily as an inhibitor of the NF-kappa-B-repressing factor (NKRF), thereby suppressing NF-κB activity. Mechanistically, it acts as a DNA methyltransferase inhibitor and induces apoptosis in cancer cells via the mitochondrial pathway, activating caspase-3 and -9, reducing anti-apoptotic proteins such as XIAP, survivin, and Mcl-1, increasing Bax levels, decreasing the Bcl-2/Bax ratio, and inhibiting p65 nuclear localization. Uroacitides has demonstrated efficacy in hematological malignancies like multiple myeloma and is used clinically for breast cancer, myelodysplastic syndromes (MDS), and non-small cell lung cancer (NSCLC). It was first approved in China in 2004.
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