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AC710 is a potent, orally bioavailable small-molecule multi-kinase inhibitor originally developed by Ambit Biosciences (now part of Daiichi Sankyo). It primarily targets the platelet-derived growth factor receptors alpha and beta (PDGFRα and PDGFRβ), as well as other type III receptor tyrosine kinases including CSF1R (FMS), KIT, and FLT3. In preclinical studies, AC710 has demonstrated significant efficacy in inhibiting the growth and migration of glioblastoma cells and glioblastoma stem cells, particularly when used in combination with inhibitors of the EGFR or mTOR pathways. Its mechanism involves blocking the ATP-binding site of its target kinases, thereby preventing downstream signaling through the PI3K/Akt/mTOR and MAPK pathways which are frequently dysregulated in genetically heterogeneous tumors.
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