Disrupting the CD47-SIRPα anti-phagocytic axis by a humanized anti-CD47 antibody is an efficacious treatment for malignant pediatric brain tumors

S Gholamin, SS Mitra, AH Feroze, J Liu… - Science translational …, 2017 - science.org
S Gholamin, SS Mitra, AH Feroze, J Liu, SA Kahn, M Zhang, R Esparza, C Richard…
Science translational medicine, 2017science.org
Morbidity and mortality associated with pediatric malignant primary brain tumors remain high
in the absence of effective therapies. Macrophage-mediated phagocytosis of tumor cells via
blockade of the anti-phagocytic CD47-SIRPα interaction using anti-CD47 antibodies has
shown promise in preclinical xenografts of various human malignancies. We demonstrate
the effect of a humanized anti-CD47 antibody, Hu5F9-G4, on five aggressive and
etiologically distinct pediatric brain tumors: group 3 medulloblastoma (primary and …
Morbidity and mortality associated with pediatric malignant primary brain tumors remain high in the absence of effective therapies. Macrophage-mediated phagocytosis of tumor cells via blockade of the anti-phagocytic CD47-SIRPα interaction using anti-CD47 antibodies has shown promise in preclinical xenografts of various human malignancies. We demonstrate the effect of a humanized anti-CD47 antibody, Hu5F9-G4, on five aggressive and etiologically distinct pediatric brain tumors: group 3 medulloblastoma (primary and metastatic), atypical teratoid rhabdoid tumor, primitive neuroectodermal tumor, pediatric glioblastoma, and diffuse intrinsic pontine glioma. Hu5F9-G4 demonstrated therapeutic efficacy in vitro and in vivo in patient-derived orthotopic xenograft models. Intraventricular administration of Hu5F9-G4 further enhanced its activity against disseminated medulloblastoma leptomeningeal disease. Notably, Hu5F9-G4 showed minimal activity against normal human neural cells in vitro and in vivo, a phenomenon reiterated in an immunocompetent allograft glioma model. Thus, Hu5F9-G4 is a potentially safe and effective therapeutic agent for managing multiple pediatric central nervous system malignancies.
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