FTO-43N

FTO-43N is a novel, selective small-molecule inhibitor of the m6A demethylase FTO (Fat mass and obesity-associated protein). Chemically characterized as an oxetane derivative, FTO-43N functions as a competitive inhibitor that selectively targets FTO over its homolog ALKBH5. By inhibiting FTO, the compound prevents the demethylation of N6-methyladenosine (m6A) and N6,2’-O-dimethyladenosine (m6Am), thereby modulating mRNA stability and translation pathways that are critical for the maintenance of cancer stem cells. In preclinical models of glioblastoma (GBM), FTO-43N has demonstrated potent antiproliferative effects and the ability to impair the self-renewal of glioma stem cells (GSCs). With favorable brain-to-plasma pharmacokinetic profiles and in vitro safety data, FTO-43N is being developed as a potential therapeutic for GBM and other malignancies driven by m6A dysregulation.