Single-cell RNA-seq of AML patient bone marrow treated with INCB059872, azacitidine, or combination

INCB059872 is a selective irreversible inhibitor of Lysine-Specific Demethylase 1 (LSD1) that is in phase 1 clinical trials in hematopoietic malignancies. We evaluated a pre-treatment bone marrow sample of a patient who showed a clinical response to INCB059872 + azacitidine treatment. Single-cell RNA-sequencing (scRNA-seq) showed that INCB059872 caused a shift in gene expression that was associated with GFI1/GFI1B regulation. A pre-INCB059872 treatment bone marrow sample from an AML patient was divided and treated ex vivo in duplicate with vehicle, INCB059872, AZA, or INCB059872 + AZA.