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Acute myeloid leukemia (AML) is the most aggressive type of blood cancer, and there is a continued need for new treatments that are well tolerated and improve long-term survival rates in patients. Induction of differentiation has emerged as a promising alternative to conventional cytotoxic chemotherapy, but known agents lack efficacy in genetically distinct patient populations. Previously, we established a phenotypic screen to identify small molecules that could stimulate differentiation in a range of AML cell lines. Utilising this strategy, a 1,5-dihydrobenzo[e][1,4]oxazepin-2(3H)-one hit compound was identified. Herein, we report the hit validation in vitro, structure-activity relationship (SAR) studies and the pharmacokinetic profiles for selected compounds.

Original publication

DOI

10.3390/molecules26216648

Type

Journal article

Journal

Molecules

Publication Date

02/11/2021

Volume

26

Keywords

CD11b, acute myeloid leukemia, benzooxazepinones, differentiation, phenotypic screen, Antineoplastic Agents, Cell Differentiation, Cell Line, Tumor, Cells, Cultured, Chemistry Techniques, Synthetic, Dose-Response Relationship, Drug, Drug Screening Assays, Antitumor, Humans, Leukemia, Myeloid, Acute, Molecular Structure, Structure-Activity Relationship