Targeting the PD-1 pathway in pediatric solid tumors and brain tumors

Lars M. Wagner, Val R. Adams

Research output: Contribution to journalReview articlepeer-review

32 Scopus citations


While remarkable advances have been made in the treatment of pediatric leukemia over the past decades, new therapies are needed for children with advanced solid tumors and high-grade brain tumors who fail standard chemotherapy regimens. Immunotherapy with immune checkpoint inhibitors acting through the programmed cell death-1 (PD-1) pathway has shown efficacy in some chemotherapy-resistant adult cancers, generating interest that these agents may also be helpful to treat certain refractory pediatric malignancies. In this manuscript we review current strategies for targeting the PD-1 pathway, highlighting putative biomarkers and the rationale for investigation of these drugs to treat common pediatric tumors such as sarcoma, neuroblastoma, and high-grade glioma. We summarize the completed and ongoing clinical trial data available, and suggest potential applications for further study.

Original languageEnglish
Pages (from-to)2097-2106
Number of pages10
JournalOncoTargets and Therapy
StatePublished - Apr 12 2017

Bibliographical note

Publisher Copyright:
© 2017 Wagner and Adams.


  • Glioma
  • Neuroblastoma
  • Nivolumab
  • PD-1
  • Pediatric
  • Pembrolizumab
  • Sarcoma

ASJC Scopus subject areas

  • Oncology
  • Pharmacology (medical)


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