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

Authors Wagner LM, Adams VR

Received 18 January 2017

Accepted for publication 8 March 2017

Published 12 April 2017 Volume 2017:10 Pages 2097—2106

DOI https://doi.org/10.2147/OTT.S124008

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Norbert Ajeawung

Peer reviewer comments 2

Editor who approved publication: Dr Yao Dai

Lars M Wagner,1 Val R Adams2

1Division of Pediatric Hematology/Oncology, 2Department of Pharmacy Practice and Science, University of Kentucky, Lexington, KY, USA

Abstract: 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.

Keywords: PD-1, nivolumab, pembrolizumab, pediatric, sarcoma, neuroblastoma, glioma

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