granulocyte-macrophage colony-stimulating factor-secreting autologous leukemia cell vaccine

A patient-specific therapeutic cancer vaccine produced by isolating autologous leukemia cells (myeloblasts) from the patient, genetically modifying them ex vivo to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF) via transduction with a replication-defective adenoviral vector encoding human GM-CSF, and then lethally irradiating the modified cells to prevent proliferation. This approach, known as GVAX, is designed to induce anti-leukemic immunity by stimulating the recruitment, differentiation, and activation of dendritic cells and other antigen-presenting cells, thereby enhancing antigen presentation and activating both cellular and humoral immune responses against the patient’s leukemia antigens. Used after allogeneic hematopoietic stem cell transplantation (HSCT) in myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML), the vaccine aims to reduce relapse by promoting a stronger graft-versus-leukemia effect with durable and specific immune responses, although randomized trials to date have shown mixed efficacy and feasibility challenges[1].

Other names

GM-CSF-secreting autologous leukemia cell vaccineGM-CSF-secreting tumor cell vaccinegranulocyte-macrophage colony-stimulating factor-secreting (GM-CSF) autologous myeloblast vaccineGM-CSF GVAX