GCT-007

GCT-007 is a brain-penetrant, small-molecule inhibitor of the phosphatidylinositol 3-kinase alpha isoform (PI3Kα, PIK3CA) being developed by Global Cancer Technology for high-grade malignancies and immune-related indications, with a particular focus on glioblastoma, diffuse intrinsic pontine glioma/diffuse midline glioma, and other pediatric high-grade gliomas.[5][6][8] Preclinical data show that GCT-007 is a highly selective p110α inhibitor that potently suppresses PI3K/AKT/mTOR signaling, inhibits tumor growth in vitro and in vivo, and crosses the blood–brain barrier, positioning it as a targeted therapy for brain tumors.[2][5][6][8] In multiple models, GCT-007 increases tumor immunogenicity (including upregulation of CD80 and modulation of PD-L1), converts “cold” tumors to “hot,” and demonstrates strong synergy with immune checkpoint inhibitors, as well as with other targeted agents such as mTOR and PKCβ inhibitors, thereby enhancing antitumor immunity and potentiating radiotherapy and endocrine therapy in resistant breast cancer models.[1][2][4][7][9] Beyond oncology, GCT-007 has generated encouraging preclinical data in psoriasis models, reflecting broader interest in PI3K pathway modulation in inflammatory and immune-mediated diseases.[6][10][14]

Other names

GCT007GCT-007GCT 007