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Review
. 2025 Jul 25;32(8):417.
doi: 10.3390/curroncol32080417.

Small-Molecule Drugs in Pediatric Neuro-Oncology

Stephanie Vairy  1   2 , George Michaiel  3   4
Affiliations
Review

Small-Molecule Drugs in Pediatric Neuro-Oncology

Stephanie Vairy et al. Curr Oncol. .

Abstract

Advances in molecular diagnostics have enabled precision medicine approaches in pediatric neuro-oncology, with small-molecule drugs emerging as promising therapeutic candidates targeting specific genetic and epigenetic alterations in central nervous system (CNS) tumors. This review provides a focused overview of several small-molecule agents under investigation or in early clinical use, including ONC201, tazemetostat, vorasidenib, CDK inhibitors, selinexor, and aurora kinase A inhibitors, among others. Highlighted are their mechanisms of action, pharmacokinetic properties, early efficacy data, and tolerability in pediatric populations. Despite encouraging preclinical and early-phase results, most agents face limitations due to study heterogeneity, lack of large-scale pediatric randomized trials, and challenges in drug delivery to the CNS. The review underscores the critical need for robust prospective clinical trials for the integration of these therapies into pediatric neuro-oncology care.

Keywords: brain tumor; pediatric neuro-oncology; targeted therapy.

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Conflict of interest statement

The authors declare no conflicts of interest.

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