EYA1 inhibitor

EYA1 (Eyes Absent Homolog 1) is a dual-function protein that acts as both a transcriptional coactivator and a protein tyrosine phosphatase. While primarily active during embryonic development, its aberrant re-expression is implicated in several cancers, most notably Mixed Lineage Leukemia-rearranged (MLL-r) leukemias. EYA1 inhibitors are small molecule agents designed to block the phosphatase activity of EYA1, which specifically targets the Tyr1 residue of the RNA polymerase II C-terminal domain (CTD). By inhibiting this dephosphorylation, these agents increase RNA Pol II CTD Tyr1 phosphorylation levels, which in turn triggers leukemia cell differentiation, G0/G1 cell cycle arrest, and cellular senescence. Preclinical research, particularly from Loyola University Chicago, has demonstrated that EYA1 inhibition reduces cell viability in human AML cell lines and significantly decreases disease progression while increasing survival in murine models of MLL-AF9 leukemia.

Other names

EYA1 inhibitorsEYA-1 inhibitorsEYA 1 inhibitors