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Advances in targeted therapies for pediatric tumors

Jia-Yi Liu1, Dong-Liang Yang1, Hai-Yan Liu1

  • 1Institute of Pharmacology and Toxicology, Zhejiang Province Key Laboratory of Anti-Cancer Drug Research, College of Pharmaceutical Sciences, Zhejiang University, Hangzhou, 310058, China.

Acta Pharmacologica Sinica
|May 17, 2026
PubMed

Insights

Pediatric tumors have unique molecular features distinct from adult cancers. Understanding these characteristics is key to developing targeted therapies for better childhood cancer treatment.

Area of Science:

  • Oncology
  • Pediatric Medicine
  • Molecular Biology

Background:

  • Pediatric tumors are a leading cause of childhood mortality, differing significantly from adult cancers.
  • They possess unique molecular profiles, including fewer mutations but more chromosomal alterations and dysregulated developmental genes.
  • These molecular features present distinct therapeutic vulnerabilities.

Purpose of the Study:

  • To review the molecular characteristics and oncogenic drivers of pediatric tumors.
  • To synthesize recent advances in targeted therapeutic strategies for pediatric malignancies.
  • To advocate for a biology-driven approach to accelerate pediatric cancer therapy development.

Main Methods:

  • Review of molecular characteristics and oncogenic drivers in pediatric tumors.
  • Systematic synthesis of recent targeted therapeutic strategies.
  • Categorization of therapies by disease type and oncogenic driver events.

Main Results:

  • Pediatric tumors exhibit unique molecular architectures, including gene rearrangements and amplification.
  • Targeted strategies include oncofusion inhibitors, agents for amplified/mutated genes, differentiation induction, antibody therapy, and cellular therapy.
  • Both pediatric-specific and adapted adult therapies show promise, but clinical translation challenges persist.

Conclusions:

  • Targeted therapies for pediatric tumors are advancing, driven by their unique biology.
  • A biology-driven framework is essential for developing effective treatments.
  • Continued research and clinical translation are crucial for improving outcomes in pediatric cancer.
Keywords:
developmental genegene amplificationmolecular characteristicsoncogenic fusionpediatric tumortargeted therapy

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