Recent research progress of Menin inhibitors in NPM1-mutated acute myeloid leukemia

VernacularTitle:Menin抑制剂治疗NPM1突变急性髓系白血病的研究进展
Author: Xiaoda YU ¹ ; Jiajing LI ; Anan WANG ; Jiangang GUO ; Bei LIU
Author Information

1. 兰州大学第一临床医学院,兰州 730000,甘肃
Publication Type:Journal Article
Keywords: acute myeloid leukemia; Menin inhib-itors; NPM1 mutation; Menin-KMT2A complex
From: Chinese Journal of Clinical Pharmacology and Therapeutics 2025;30(4):533-540
CountryChina
Language:Chinese
Abstract: The nucleophosmin 1(NPM1)muta-tion is one of the most frequent subtypes in acute myeloid leukemia(AML).Under the conditions of FLT3-internal tandem duplications(FLT3-ITD)and/or DNMT3A co-mutations or adverse cytogenetics,the originally favorable prognosis will deteriorate.In recent years,studies have found that multiple endocrine neoplasia protein(Menin)inhibitors tar-geting Menin-KMT2A complex can downregulate the overexpression of leukemia causing genes HOX(homeotic gene)and MEIS1(myeloid ecotropic vi-ral integration site 1)in NPM1-mutated AML,dem-onstrating remarkable anti-leukemia activity.This article aims to review the mechanism and clinical research of Menin inhibitors,novel small molecule targeted drugs in NPM1-mutated AML,as well as the resistance mechanism of Menin inhibitors,hop-ing to provide promising approaches for the subse-quent treatment of NPM1-mutated AML patients.