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New and Emerging Targeted Therapies for Pediatric Acute Myeloid Leukemia (AML)

Jing Chen1, Chana L Glasser2

  • 1Division of Pediatric Hematology/Oncology, Hackensack University Medical Center, Hackensack, NJ 07601, USA.

Children (Basel, Switzerland)
|February 14, 2020
PubMed

Insights

Relapsed acute myeloid leukemia (AML) in children has poor outcomes. New targeted therapies, including antibody drug conjugates and small molecule inhibitors, offer hope for treating relapsed/refractory AML.

Area of Science:

  • Pediatric Oncology
  • Hematology
  • Pharmacology

Background:

  • Relapse rates in pediatric acute myeloid leukemia (AML) remain high despite treatment advances.
  • Prognosis for children with relapsed/refractory AML is poor, with chemotherapy intensification reaching its limits due to toxicity.

Purpose of the Study:

  • To review novel therapeutic agents for pediatric relapsed/refractory AML.
  • To highlight antibody drug conjugates (ADCs), small molecule inhibitors, and tyrosine kinase inhibitors in early clinical development.

Main Methods:

  • Literature review of recent advancements in AML biology and targeted therapies.
  • Focus on drugs targeting specific molecular pathways in AML.
  • Emphasis on agents in early phase clinical trials for pediatric patients.

Main Results:

  • Several new targeted therapies are emerging for pediatric AML.
  • Antibody drug conjugates (ADCs), small molecule inhibitors, and tyrosine kinase inhibitors show promise.
  • Many of these novel agents are progressing through early-phase clinical trials.

Conclusions:

  • Improved understanding of AML biology has led to targeted drug development.
  • Novel therapies offer a promising alternative for children with relapsed/refractory AML.
  • Clinical evaluation of these agents is crucial for improving outcomes.

Keywords:
acute myeloid leukemiaantibody drug conjugate

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