naxitamab + sargramostim + irinotecan + temozolomide

This combination regimen consists of **naxitamab** (a humanized monoclonal antibody that targets the disialoganglioside GD2), **sargramostim** (granulocyte-macrophage colony-stimulating factor, GM-CSF), **irinotecan** (a topoisomerase I inhibitor), and **temozolomide** (an oral alkylating agent). The combination is primarily used for the treatment of **high-risk neuroblastoma**, especially relapsed/refractory cases. Naxitamab mediates its antitumor activity against GD2-expressing neuroblastoma cells via antibody-dependent cell-mediated cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC). Sargramostim stimulates the production and activation of granulocytes and macrophages, augmenting immune responses and possibly enhancing the efficacy of naxitamab. Irinotecan inhibits DNA replication by stabilizing topoisomerase I-DNA complexes, causing DNA strand breaks. Temozolomide is a DNA-alkylating agent that damages tumor cell DNA, leading to apoptosis. This combination is delivered in treatment cycles and is recommended by the NCCN for relapsed/refractory high-risk neuroblastoma based on both clinical trial and compassionate access data.

Other names

sargramostim (for GM-CSF)