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AZ32 is an orally bioavailable, small molecule inhibitor of the ataxia-telangiectasia mutated (ATM) kinase, originally developed by AstraZeneca. It is specifically designed to penetrate the blood-brain barrier (BBB), making it a candidate for treating central nervous system malignancies such as glioblastoma multiforme (GBM). By inhibiting ATM, a key sensor in the DNA damage response (DDR) pathway, AZ32 acts as a radiosensitizer, enhancing the lethal effects of ionizing radiation on tumor cells. Preclinical studies have demonstrated its efficacy in orthotopic mouse glioma models and its ability to reverse multidrug resistance in colorectal cancer by inhibiting the ABCG2 transporter. The compound is currently in the research phase and has not yet been approved for clinical use.
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