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Topotecan is a semi-synthetic, water-soluble derivative of camptothecin, classified as a topoisomerase I inhibitor. It is an antineoplastic (chemotherapy) agent used primarily for the treatment of ovarian cancer, small cell lung cancer, and cervical cancer. Topotecan works by inhibiting the enzyme topoisomerase I, which is essential for DNA replication and repair. By stabilizing the complex between DNA and topoisomerase I after single-strand breaks are introduced during DNA replication, it prevents religation of these breaks. This leads to accumulation of double-stranded DNA damage when replication forks collide with these complexes, ultimately resulting in apoptosis (programmed cell death) in rapidly dividing tumor cells. Topotecan may also induce oxidative stress and inhibit hypoxia-inducible factor pathways. It is available as oral capsules or intravenous infusion under the brand name Hycamtin.
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