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Carmofur is a small molecule antineoplastic agent and a derivative of fluorouracil (5-FU), primarily used in the treatment of colorectal and breast cancer. It acts as a prodrug that is converted intracellularly to 5-FU but also exerts direct pharmacological effects by inhibiting acid ceramidase (AC), an enzyme involved in ceramide metabolism that influences cancer cell survival and death. Inhibition of AC sensitizes tumor cells to chemotherapy and radiation. Carmofur has also been shown to inhibit thymidylate synthase, preventing DNA replication. Additionally, it has demonstrated activity against the SARS-CoV-2 main protease in preclinical studies, suggesting potential antiviral applications. Carmofur is approved for use in Japan but has not been FDA-approved in the United States[1][2][4][5][6].
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