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Targeted therapy in KMT2Ar AML

Ying Zhang1, Yankun Yang1, Yiwen Du1

  • 1Department of Hematology, West China Hospital, Sichuan University, Chengdu, People's Republic of China.

Hematology (Amsterdam, Netherlands)
|December 10, 2025
PubMed
Abstract

Insights

Targeted therapies show promise for acute myeloid leukemia (AML) with KMT2A rearrangement (KMT2Ar), a subtype resistant to chemotherapy. New agents, including menin inhibitors, are under investigation to improve patient outcomes for this challenging leukemia.

Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Background:

  • Acute myeloid leukemia (AML) with KMT2A rearrangement (KMT2Ar) presents a significant clinical challenge due to chemotherapy resistance and poor prognosis.
  • Understanding the molecular intricacies of KMT2Ar AML is crucial for developing effective therapeutic strategies.

Purpose of the Study:

  • To review current advancements in targeted therapy for KMT2Ar AML.
  • To highlight potential therapeutic strategies and drugs in clinical development for KMT2Ar AML.

Main Methods:

  • Comprehensive review of studies focusing on the molecular characteristics of KMT2Ar AML.
  • Analysis of targeted drugs affecting key genetic and epigenetic mechanisms in KMT2Ar AML.
  • Collection and examination of data on drug mechanisms, preclinical findings, and clinical trials.

Main Results:

  • Emerging targeted agents, such as menin inhibitors, demonstrate encouraging clinical activity in KMT2Ar AML.
  • Inhibitors targeting DOT1L, BET, and EZH2 pathways show promising preclinical results.
  • Early evidence suggests combination therapies may be superior to monotherapy in overcoming drug resistance.

Conclusions:

  • Targeted therapy offers a promising new direction for KMT2Ar AML by addressing abnormal molecular networks.
  • Current therapies are largely in early developmental stages with limited clinical translation.
  • Further research is imperative to enhance treatment safety and long-term efficacy for KMT2Ar AML patients.
Keywords:
AMLKMT2Armenin inhibitorstargeted therapy

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