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Predictive Biomarkers in Oncology pp 375–382Cite as

Targeted Therapies for Pediatric Central Nervous System Tumors

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Abstract

Recent discoveries have significantly enhanced our understanding of the biology of pediatric central nervous system tumors. Molecularly defined targeted therapies are now being used to treat subgroups of these tumors, mostly in the setting of clinical trials. These therapies include targeted inhibition of smoothened to treat sonic hedgehog medulloblastoma; of mammalian target of rapamycin to treat subependymal giant cell astrocytoma; and of the mitogen-activated protein kinase pathway to treat BRAF V600E-mutated low-grade and high-grade gliomas, KIAA1549:BRAF fusion-positive pilocytic astrocytoma, and plexiform neurofibroma. For many patients, the use of these targeted therapies has resulted in significant regression and/or improved control of their tumors, including tumors that are recurrent or refractory to conventional therapy.

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Abbreviations

CNS:

Central nervous system

HGG:

High-grade glioma

LGG:

Low-grade glioma

MAPK:

Mitogen-activated protein kinase

MEK:

Mitogen-activated extracellular signal-regulated kinase

mTOR:

Mammalian target of rapamycin

NF1:

Neurofibromatosis type 1

PN:

Plexiform neurofibroma

PTCH1:

Patched 1

SEGA:

Subependymal giant cell astrocytoma

SHH:

Sonic hedgehog

SMO:

Smoothened

SUFU:

Suppressor of fused

TSC:

Tuberous sclerosis complex

WHO:

World Health Organization

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Author information

Authors and Affiliations

  1. Division of Hematology/Oncology, Department of Pediatrics, University of Utah and Primary Children’s Hospital, Salt Lake City, UT, USA

    Nicholas Shawn Whipple

  2. Department of Oncology, St. Jude Children’s Research Hospital, Memphis, TN, USA

    Amar Gajjar

Authors
  1. Nicholas Shawn Whipple
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  2. Amar Gajjar
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Corresponding author

Correspondence to Nicholas Shawn Whipple .

Editor information

Editors and Affiliations

  1. Department of Pathology and Lab Medicine, Indiana University School of Medicine, Indianapolis, IN, USA

    Sunil Badve

  2. Targos Inc., Issaquah, WA, USA

    George Louis Kumar

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Whipple, N.S., Gajjar, A. (2019). Targeted Therapies for Pediatric Central Nervous System Tumors. In: Badve, S., Kumar, G. (eds) Predictive Biomarkers in Oncology. Springer, Cham. https://doi.org/10.1007/978-3-319-95228-4_33

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  • DOI: https://doi.org/10.1007/978-3-319-95228-4_33

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  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-319-95227-7

  • Online ISBN: 978-3-319-95228-4

  • eBook Packages: MedicineMedicine (R0)

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