PURPOSE OF REVIEW This article focuses on primary brain tumors in the pediatric population with an emphasis on molecular classifications and treatment strategies.

RECENT FINDINGS Pediatric brain tumors are a heterogeneous group of tumors that differ from adult brain cancers despite similar nomenclature. With the added complexity of the developing brain, treatment regimens are tailored to protect neurocognitive outcomes without sacrificing long-term survival. The 2016 World Health Organization’s classification incorporated molecular characteristics to aid in defining the diagnosis and prognosis of these tumors. These changes have enabled providers to stratify patients, thus intensifying therapies in those with high-risk diseases and modifying treatments to reduce morbidity for children and to provide better outcomes. Recent published findings from clinical trials have been especially helpful for gliomas, embryonal tumors, and ependymomas. By using this new information, molecular factors that correlate with survival have been identified in patients. In addition, genetic findings in tumor tissue have also led to revelations in predisposing germline mutations.

SUMMARY New findings from clinical trials and molecular stratification will shape the next generation of therapies in hopes of improving overall outcome, identifying pathways in tumorigenesis, and aiding in genetic counseling for children and their families.

Address correspondence to Dr Michelle Monje, 265 Campus Dr, Room G3077, Stanford, CA 94305, [email protected].

RELATIONSHIP DISCLOSURE: Dr Partap has served on advisory boards for Bayer AG and Fennec Pharmaceuticals and as a consultant for Gerson Lehrman Group, Inc. Dr Partap has received personal compensation for speaking engagements from the American Academy of Neurology, the American Academy of Pediatrics, the Medical Education Speakers Network, and the Society for Neuro-Oncology and research/grant support from AbbVie Inc and the National Institutes of Health/National Cancer Institute (5 UM1 CA081457 18). Dr Monje serves on the editorial advisory boards for Cancer Cell, Neuro-Oncology, and Neuron; has received personal compensation for serving on the scientific advisory board for Cygnal Therapeutics; and has received research/grant support from Cancer Research UK, the National Cancer Institute (P50CA165962), the National Institute of Neurological Disorders and Stroke (R01NS092597), the National Institutes of Health (DP1NS111132), and the Robert J. Kleberg Jr and Helen C. Kleberg Foundation.

UNLABELED USE OF PRODUCTS/INVESTIGATIONAL USE DISCLOSURE: Drs Partap and Monje discuss the unlabeled/investigational use of medications for pediatric brain tumors, none of which are approved by the US Food and Drug Administration.