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This investigational combination therapy consists of pucotenlimab, lenvatinib, and irinotecan, and is currently being evaluated in a Phase II clinical trial (NCT07188311) for the treatment of advanced, relapsed, or refractory hepatoblastoma in pediatric patients. Pucotenlimab is a humanized monoclonal antibody that acts as an immune checkpoint inhibitor by targeting the programmed cell death protein 1 (PD-1) receptor, thereby enhancing T-cell mediated anti-tumor activity. Lenvatinib is an oral multi-kinase inhibitor that targets vascular endothelial growth factor receptors (VEGFR1-3), fibroblast growth factor receptors (FGFR1-4), platelet-derived growth factor receptor alpha (PDGFRα), KIT, and RET, primarily inhibiting tumor angiogenesis and proliferation. Irinotecan is a cytotoxic chemotherapy agent that inhibits DNA topoisomerase I, leading to double-strand DNA breaks and cell death. This triplet regimen represents a multi-modal approach combining immunotherapy, targeted anti-angiogenic therapy, and traditional chemotherapy for rare pediatric liver cancers.
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