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BAU-243 is a novel small molecule BCL-2 inhibitor being developed for the treatment of glioblastoma (GBM). Identified through a multistep virtual screening of over 210,000 compounds, BAU-243 demonstrates high affinity for the B-cell leukemia/lymphoma-2 (BCL-2) protein. Unlike selective BCL-2 inhibitors such as venetoclax (ABT-199) which primarily induce apoptosis, BAU-243 induces autophagic cell death in GBM cells, including cancer-initiating cells, by disrupting the Beclin 1:Bcl-2 complex. In preclinical orthotopic brain tumor models, BAU-243 has shown a significant reduction in tumor growth compared to vehicle and venetoclax-treated groups.
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