Advances in menin inhibition in acute myeloid leukemia Journal Article


Authors: Boussi, L.; Cai, S. F.; Stein, E. M.
Article Title: Advances in menin inhibition in acute myeloid leukemia
Abstract: Menin has emerged as a promising therapeutic target in acute myeloid leukemia (AML). The menin–MLL1 interaction promotes an oncogenic transcriptional program that drives leukemogenesis in HOX-mediated acute leukemias, including KMT2A-rearranged (KMT2Ar), nucleophosmin 1–mutated (NPM1m), and NUP98-rearranged (NUP98r) AML, prompting development of menin inhibitors for treatment of these subtypes. Successes in clinical investigation have led to recent FDA approval of revumenib for KMT2Ar AML, with numerous trials examining menin inhibitors as monotherapy and in combination with other antileukemic drugs ongoing. Although menin inhibitors represent a major advancement in AML treatment, acquired resistance is an evolving barrier to efficacy. Here, we examine the biological rationale for menin inhibition and discuss the landscape of clinical trials and resistance mechanisms associated with menin inhibitors. © 2025 Elsevier Inc.
Keywords: acute myeloid leukemia; kmt2a; npm1; menin inhibitors
Journal Title: Trends in Cancer
ISSN: 2405-8025
Publisher: Cell Press  
Publication status: Online ahead of print
Date Published: 2025-01-01
Online Publication Date: 2025-01-01
Language: English
DOI: 10.1016/j.trecan.2025.06.002
PROVIDER: scopus
DOI/URL:
Notes: Review -- Source: Scopus
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