Molecularly Targeted Agents in the Therapy of Pediatric Brain Tumors

Miriam Bornhorst, Eugene I. Hwang*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

7 Scopus citations


Pediatric brain tumors are the leading cause of cancer-related death in children. Recent advances in sequencing techniques, and collaborative efforts to encode the mutational landscape of various tumor subtypes, have resulted in the identification of recurrent mutations that may present as actionable targets in these tumors. A number of molecularly targeted agents are approved or in development for the treatment of various tumor types in adult patients. Similarly, these agents are increasingly being incorporated into pediatric clinical trials, allowing for a targeted approach to treatment. However, due to the genetic heterogeneity of these tumors, focused clinical trials in pediatric patients are challenging and regulatory hurdles may delay access to therapeutic compounds that are in regular use in adult patients. The tumor site-agnostic clinical development of TRK inhibitors for pediatric solid tumors is a current example of how the combination of genetic testing and innovative clinical trial design can accelerate the clinical development of targeted agents for pediatric patients.

Original languageEnglish (US)
Pages (from-to)45-54
Number of pages10
JournalPediatric Drugs
Issue number1
StatePublished - Feb 1 2020

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pharmacology (medical)


Dive into the research topics of 'Molecularly Targeted Agents in the Therapy of Pediatric Brain Tumors'. Together they form a unique fingerprint.

Cite this