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Related Experiment Video

Updated: Feb 28, 2026

Intracellular Phosphoflow Cytometry of Acute Myeloid Leukemia Patient-Derived Xenotransplants
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Menin Inhibition in Acute Myeloid MLL Rearranged Leukemias: A New Target for Precision Care.

Caterina Alati1, Matteo Molica2, Martina Pitea1

  • 1Hematology and Stem Cell Transplantation and Cellular Therapies Unit (CTMO), Department of Hemato-Oncology and Radiotherapy, Grande Ospedale Metropolitano "Bianchi-Melacrino-Morelli", 89133 Reggio Calabria, Italy.

Cancers
|February 27, 2026
PubMed
Summary

Menin inhibitors offer new hope for high-risk acute leukemias. These targeted therapies show promise in clinical trials, with some patients achieving remission and minimal residual disease negativity.

Keywords:
KMT2A rearrangementsMLL rearrangementsNPM1 mutationsacute myeloid leukemiamenin inhibitorsprecision medicinerevumenibtargeted therapy

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Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Background:

  • KMT2A-rearranged and NPM1-mutated acute leukemias represent high-risk subtypes with significant unmet therapeutic needs.
  • Menin inhibitors have emerged as the first targeted therapies for these specific leukemias.

Purpose of the Study:

  • To review the molecular mechanisms of the menin-KMT2A interaction.
  • To summarize clinical trial data on the efficacy and safety of menin inhibitors in monotherapy and combination settings.

Main Methods:

  • Review of clinical trial data for menin inhibitors.
  • Analysis of molecular mechanisms of menin-KMT2A interaction and resistance.

Main Results:

  • Revumenib shows a 23% composite complete remission rate in heavily pretreated patients, with 61% MRD negativity.
  • Multiple menin inhibitors are in development with varying safety profiles.
  • Combination therapies demonstrate high response rates in newly diagnosed patients.
  • Acquired resistance occurs in ~40% of cases, often due to MEN1 mutations.

Conclusions:

  • Menin inhibitors represent a significant advancement in treating KMT2A-rearranged and NPM1-mutated acute leukemias.
  • Combination therapies and sequential treatments may overcome resistance and improve outcomes.
  • Allogeneic transplantation remains a crucial curative option for eligible patients.