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REVIEW article

Front. Oncol.
Sec. Pediatric Oncology
Volume 15 - 2025 | doi: 10.3389/fonc.2025.1504803
This article is part of the Research Topic Recent Biological Insights into Pediatric Brain Tumors View all 9 articles

Targeted Therapy in Pediatric Central Nervous System Tumors: A Review from the National Pediatric Cancer Foundation

Provisionally accepted
Benjamin I Siegel Benjamin I Siegel 1*Prabhumallikarjun Patil Prabhumallikarjun Patil 2,3Akul Prakash Akul Prakash 4Darren Klawinski Darren Klawinski 5Eugene I Hwang Eugene I Hwang 1
  • 1 Children’s National Hospital, Washington D.C., United States
  • 2 Children's Healthcare of Atlanta, Atlanta, Georgia, United States
  • 3 School of Medicine, Emory University, Atlanta, Georgia, United States
  • 4 New York University, New York City, New York, United States
  • 5 Nemours Children's Hospital, Orlando, Florida, United States

The final, formatted version of the article will be published soon.

    Central nervous system tumors represent the leading cause of cancer-related mortality in children. Conventional therapies of surgery, radiation, and cytotoxic chemotherapy have insufficient efficacy for some pediatric CNS tumors and are associated with significant morbidity, prompting an ongoing need for novel treatment approaches. Identification of molecular alterations driving tumorigenesis has led to a rising interest in developing targeted therapies for these tumors. The present narrative review focuses on recent progress in targeted therapies for pediatric CNS tumors. We outline the key implicated cellular pathways, discuss candidate molecular therapies for targeting each pathway, and present an overview of the clinical trial landscape for targeted therapies in pediatric CNS tumors. We then discuss challenges and future directions for targeted therapy, including combinatorial approaches and real-time drug screening for personalized treatment planning.

    Keywords: Neuro-Oncology, targeted therapy, MAPK, mTOR, epigenetics, brain tumors, VEGF, receptor tyrosine kinase

    Received: 01 Oct 2024; Accepted: 23 Jan 2025.

    Copyright: © 2025 Siegel, Patil, Prakash, Klawinski and Hwang. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Benjamin I Siegel, Children’s National Hospital, Washington D.C., United States

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.