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December 8, 2025BloodOpen Access

Combined FLT3 and EZH1/2 inhibition reduces LSCs and promotes myeloid differentiation in PDX models of AML

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Authors

EWEunice S. Wang

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Overview

Combined FLT3 and EZH1/2 inhibition reduced leukemia stem cells and improved myeloid differentiation in AML, suggesting a novel therapeutic approach.

Key Points

  • To evaluate the combined effectiveness of FLT3 and EZH1/2 inhibitors in reducing leukemia stem cells and promoting myeloid differentiation in acute myeloid leukemia.
  • Utilized patient-derived xenograft (PDX) models of FLT3-ITD AML for treatment assessment.
  • Administered gilteritinib and valemetostat to models and analyzed leukemia burden and differentiation.
  • Conducted serial bone marrow assessments and single cell RNA-Seq analysis to monitor treatment effects.
  • Combination treatment significantly reduced leukemia stem cells (LSCs) in bone marrow and spleen.
  • Increased myeloid differentiation observed post-treatment, primarily noted in neutrophils and monocytes.
  • Single cell RNA-Seq analysis revealed distinct differentiation states between gilteritinib alone and combination treatments.

Cite This Study

Eunice S. Wang (2025) studied this question.

synapsesocial.com/papers/69362f604fa91c937236dcddhttps://doi.org/10.1182/blood-2025-1488
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