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Trastuzumab + sargramostim (GM-CSF) is an investigational combination immunotherapy regimen being studied for the treatment of relapsed or recurrent posterior fossa ependymoma (PF EPN) in pediatric and young adult patients. The regimen consists of trastuzumab, a monoclonal antibody targeting the Human Epidermal Growth Factor Receptor 2 (HER2), and sargramostim, a recombinant granulocyte-macrophage colony-stimulating factor (GM-CSF). In this therapeutic approach, trastuzumab is administered intrathecally to bypass the blood-brain barrier and directly target HER2-overexpressing tumor cells within the central nervous system. Sargramostim is administered subcutaneously to stimulate and recruit monocyte and macrophage effector cells, which are essential for mediating antibody-dependent cell-mediated cytotoxicity (ADCC) against the tumor cells. This combination is primarily being developed and evaluated by researchers at the University of Colorado Denver and Children's Hospital Colorado.
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