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melphalan + topotecan
- Development stage
- Phase 3
- Lead developer
- GSK
- Modality
- Small Molecules
- Administration
- Intra-arterial, Intravitreal
01
Overview
Melphalan + topotecan is a combination chemotherapy regimen consisting of two small molecule antineoplastic agents, melphalan and topotecan. Melphalan is an alkylating agent that crosslinks DNA strands, leading to inhibition of DNA and RNA synthesis and subsequent cell death. Topotecan is a topoisomerase I inhibitor that prevents the religation of single-strand breaks in DNA during replication, resulting in cytotoxicity. This combination has been primarily investigated for intra-arterial or intravitreal administration in the treatment of retinoblastoma, particularly for globe salvage therapy in children with intraocular disease. The regimen has demonstrated synergistic pharmacologic activity with manageable toxicity profiles comparable to single-agent melphalan[1][2][3].
- Other names
- MPT
02
Targets
DNATOP1 (DNA Topoisomerase I)
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