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CHMFL-FLT3-335 is a **small molecule inhibitor** specifically designed to target the **FMS-like tyrosine kinase 3 internal tandem duplication mutant (FLT3-ITD)**, which is a critical driver mutation observed in a significant subset of acute myeloid leukemia (AML) cases. The drug exhibits high selectivity for FLT3-ITD over wild-type FLT3 and cKIT, as demonstrated by its potent inhibition of proliferation in FLT3-ITD-positive AML cell lines and patient-derived primary cells, without significant effects on wild-type FLT3 primary cells. Mechanistically, CHMFL-FLT3-335 suppresses FLT3 kinase phosphorylation and its downstream signaling, induces apoptosis, and enforces cell cycle arrest in the G0/G1 phase. In preclinical xenograft models, the compound also shows robust tumor growth suppression and favorable pharmacokinetic properties. The compound was originally developed by researchers at the Chinese Academy of Sciences as a potential therapy for FLT3-ITD mutant AML[4][9][10].
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