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Advancements in targeted therapies for acute myeloid leukemia

Matthew P Connor1, Adam Barsouk1, Omar Elghawy1

  • 1University of Pennsylvania, Department of Medicine, Division of Hematology and Oncology, 3400 Civic Center Blvd, 12 South Pavilion, Philadelphia, PA 19104, USA.

Current Opinion in Pharmacology
|January 23, 2026
PubMed

Insights

Precision therapies targeting specific mutations like FLT3, IDH1/IDH2, KMT2A, and NPM1 are revolutionizing acute myeloid leukemia (AML) treatment beyond traditional chemotherapy.

Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Background:

  • Acute myeloid leukemia (AML) treatment historically relied on intensive chemotherapy.
  • Recent advances in understanding oncogenesis have enabled targeted therapies.

Purpose of the Study:

  • To review precision therapies targeting key AML mutational pathways.
  • To summarize clinical trials and future directions for targeted AML treatments.

Main Methods:

  • Review of major clinical trials for FLT3, IDH1/IDH2, KMT2A, and NPM1 inhibitors.
  • Analysis of safety and efficacy data for targeted agents.
  • Discussion of ongoing and upcoming combination therapies.

Main Results:

  • Small molecule inhibitors targeting FLT3, IDH1/IDH2, KMT2A, and NPM1 show efficacy.
  • Precision therapies offer new options for molecular subsets of AML.
  • Combination strategies are being explored for enhanced outcomes.

Conclusions:

  • Targeted therapies represent a significant advancement in AML treatment.
  • Further research into novel and combination therapies is crucial for improving patient outcomes.
  • Personalized medicine approaches are transforming AML management.

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