2470-51

2470-51 is a novel polyamine sulfonamide small molecule identified by researchers at the University of Florida as a selective therapeutic candidate for Acute Myeloid Leukemia (AML). Discovered through high-throughput chemical-phenotypic screening of over 30 million compounds, 2470-51 is designed to target AML cells sequestered within the protective bone marrow vascular niche. The compound exerts its anti-leukemic effect by covalently binding to heterogeneous nuclear ribonucleoprotein C1/C2 (hnRNPC), a splicing repressor that AML cells appear selectively dependent upon. Inhibition of hnRNPC leads to mitotic cell cycle arrest and apoptosis. Preclinical studies have demonstrated that 2470-51 selectively kills AML blasts and leukemic stem/progenitor cells while sparing normal hematopoietic stem cells and endothelial cells, showing significant efficacy in patient-derived xenograft (PDX) models.

Other names

polyamine sulfonamide 2470-51