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Small-Molecule Drugs in Pediatric Neuro-Oncology

Stephanie Vairy1,2, George Michaiel3,4

  • 1Division of Hematology-Oncology, Department of Pediatrics, Centre Hospitalier Universitaire de Sherbrooke, Université de Sherbrooke, Sherbrooke, QC J1H 5N4, Canada.

Current Oncology (Toronto, Ont.)
|August 27, 2025
PubMed

Insights

Precision medicine uses small-molecule drugs to target genetic alterations in pediatric central nervous system (CNS) tumors. Further clinical trials are crucial for integrating these promising therapies into neuro-oncology care.

Area of Science:

  • Pediatric Neuro-oncology
  • Molecular Diagnostics
  • Precision Medicine

Background:

  • Molecular diagnostics advance precision medicine for pediatric central nervous system (CNS) tumors.
  • Small-molecule drugs target specific genetic and epigenetic alterations in CNS tumors.
  • This review focuses on emerging small-molecule agents in pediatric neuro-oncology.

Purpose of the Study:

  • To provide an overview of small-molecule agents for pediatric CNS tumors.
  • To highlight mechanisms of action, pharmacokinetics, efficacy, and tolerability.
  • To identify limitations and future research needs for these therapies.

Main Methods:

  • Review of small-molecule agents under investigation or in early clinical use.
  • Focus on agents like ONC201, tazemetostat, vorasidenib, CDK inhibitors, selinexor, and aurora kinase A inhibitors.
  • Analysis of preclinical and early-phase clinical data in pediatric populations.

Main Results:

  • Several small-molecule agents show promise for pediatric CNS tumors.
  • Early data indicate varying efficacy, pharmacokinetic profiles, and tolerability.
  • Limitations include study heterogeneity, lack of large pediatric trials, and CNS drug delivery challenges.

Conclusions:

  • Small-molecule drugs represent a promising therapeutic avenue in pediatric neuro-oncology.
  • Robust prospective clinical trials are essential for clinical integration.
  • Addressing challenges in study design and drug delivery is critical for advancing care.
Keywords:
brain tumorpediatric neuro-oncologytargeted therapy

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