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GVAX leukemia vaccine is a cell-based cancer immunotherapy developed by Cell Genesys for the treatment of acute myeloid leukemia (AML). The vaccine platform utilizes a combination of irradiated autologous leukemia cells (derived from the patient) and an allogeneic bystander cell line (K562, designated CG9962) that has been genetically modified to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF). The mechanism involves the local secretion of GM-CSF at the injection site, which recruits and activates antigen-presenting cells, particularly dendritic cells. These cells then ingest the leukemia-associated antigens from the irradiated tumor cells and present them to the patient's T-cells, thereby stimulating a systemic immune response against the malignancy. In clinical trials, the vaccine was often used in the context of autologous stem cell transplantation to prime lymphocytes for reinfusion, aiming to eliminate minimal residual disease.
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