FTO regulates ELK3-mediated metabolic rewiring and represents a unique therapeutic target in T cell leukemia.

Huang, Hao; Li, Xinlu; Luo, Jinlian; Gao, Chuan; Yang, Mengjie; Xu, Jin; Xie, Ting; Chen, Zhi et al. · Sci Adv · 2025

basic_science · Level V

Where this comes from

Abstract

Understanding the regulation of N6-methyladenosine (m<sup>6</sup>A), the prominent internal modification in mRNA, fosters the development of potential therapeutic strategies for human cancers. While the m<sup>6</sup>A demethylases FTO and ALKBH5 are recognized for their crucial roles in various cancers, their impact on lymphoid leukemia remains uncertain. Using T cell acute lymphoblastic leukemia (T-ALL) as a model system, we identify FTO as a unique vulnerability in T cell leukemia. Knockout of <i>FTO</i>, but not <i>ALKBH5</i>, significantly suppresses leukemia initiation and progression. Mechanistic analysis reveals that FTO heightens <i>ELK3</i> mRNA stability in an m<sup>6</sup>A-dependent manner. Elevated ELK3 in turn transcriptionally activates the expression of glycolytic genes. Pharmacological inhibition of FTO suppresses <i>ELK3</i> expression, hampers glycolysis and manifests remarkable antileukemia efficacy. Our findings unravel the crucial role of FTO in T-ALL and highlight the FTO-ELK3 axis as a key nodule during leukemogenesis, thereby providing a fundamental basis to harness selective FTO antagonist for T-ALL therapeutics.

Medical subject headings