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This combination therapy consists of toripalimab (JS001), an anti-PD-1 monoclonal antibody; lenvatinib, a multi-kinase inhibitor; and the GEMOX chemotherapy regimen (gemcitabine and oxaliplatin). It is being investigated primarily for the treatment of advanced and unresectable intrahepatic cholangiocarcinoma (iCCA). Toripalimab works by blocking the PD-1 receptor on T-cells, enhancing the immune response against tumor cells. Lenvatinib inhibits multiple receptor tyrosine kinases, including VEGFR, FGFR, and PDGFR, to inhibit angiogenesis and tumor growth. Gemcitabine and oxaliplatin are cytotoxic agents that interfere with DNA synthesis and cause DNA damage, respectively. A Phase 2 study conducted at Shanghai Zhongshan Hospital (NCT03951597) demonstrated significant efficacy in patients with advanced iCCA, showing an objective response rate of 80%.
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