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Updated: Feb 27, 2026

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Decoding Leukemic Stem Cells in AML: From Identification to Targeted Eradication.

Elisavet Apostolidou1, Vasileios Georgoulis1, Dimitrios Leonardos1

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Diseases (Basel, Switzerland)
|February 26, 2026
PubMed
Summary
This summary is machine-generated.

Leukemic stem cells (LSCs) drive acute myeloid leukemia (AML) relapse. Understanding LSC biology and targeting these therapy-resistant cells is crucial for improving AML treatment outcomes and preventing disease recurrence.

Keywords:
acute myeloid leukemiaimmunophenotypeleukemic stem cellstargeted treatment

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Area of Science:

  • Hematology
  • Cancer Biology
  • Stem Cell Research

Background:

  • Acute myeloid leukemia (AML) presents significant therapeutic challenges due to high relapse rates.
  • Leukemic stem cells (LSCs) are a rare, therapy-resistant population responsible for disease re-initiation and relapse.

Purpose of the Study:

  • To review recent advances in LSC biology, focusing on functional and phenotypic differences from normal hematopoietic stem cells (HSCs).
  • To discuss LSC quantification methods, underlying molecular programs, and current/emerging therapeutic strategies.
  • To examine translational barriers and propose a framework for integrating LSC assessment into clinical practice.

Main Methods:

  • Synthesis of current literature on LSC biology, function, and therapeutic targeting.
  • Discussion of multiparameter flow cytometry and marker panels for LSC quantification.
  • Review of signaling, metabolic, and epigenetic dysregulation in LSCs.

Main Results:

  • LSCs exhibit distinct functional and phenotypic characteristics compared to HSCs.
  • Dysregulated cellular programs contribute to LSC persistence and measurable residual disease.
  • Various therapeutic strategies targeting LSCs are under investigation, alongside identified translational barriers.

Conclusions:

  • Targeting LSCs is essential for overcoming AML therapeutic challenges and reducing relapse rates.
  • Further research and assay harmonization are needed to effectively integrate LSC assessment into AML management.
  • Developing novel LSC-directed therapies holds promise for improving patient outcomes in AML.