Biologically targeted therapeutics in pediatric brain tumors

Amulya A. Nageswara Rao, Joseph Scafidi, Elizabeth M. Wells, Roger J. Packer

Research output: Contribution to journalReview article

Abstract

Pediatric brain tumors are often difficult to cure and involve significant morbidity when treated with traditional treatment modalities, including neurosurgery, conventional chemotherapy, and radiotherapy. During the past two decades, a clearer understanding of tumorigenesis, molecular growth pathways, and immune mechanisms in the pathogenesis of cancer has opened up promising avenues for therapy. Pediatric clinical trials with novel biologic agents are underway to treat various pediatric brain tumors, including high and low grade gliomas and embryonal tumors. As the therapeutic potential of these agents undergoes evaluation, their toxicity profiles are also becoming better understood. These agents have potentially better central nervous system penetration and loweracty toxicity profiles compared with conventional chemotherapy. In infants and younger children, biologic agents may prove to be of equal or greater efficacy compared with traditional chemotherapy and radiation therapy, and may reduce the deleterious side effects of traditional therapeutics on the developing brain. Molecular pathways implicated in pediatric brain tumors, agents that target these pathways, and current clinical trials are reviewed. Associated neurologic toxicities will be discussed subsequently. Considerable work is needed to establish the efficacy of these agents alone and in combination, but pediatric neurologists should be aware of these agents and their rationale.

Original languageEnglish (US)
Pages (from-to)203-211
Number of pages9
JournalPediatric Neurology
Volume46
Issue number4
DOIs
StatePublished - Apr 1 2012
Externally publishedYes

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Brain Neoplasms
Pediatrics
Biological Factors
Drug Therapy
Radiotherapy
Clinical Trials
Therapeutics
Neurosurgery
Therapeutic Uses
Glioma
Nervous System
Neoplasms
Carcinogenesis
Central Nervous System
Morbidity
Brain
Growth

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Developmental Neuroscience
  • Neurology

Cite this

Biologically targeted therapeutics in pediatric brain tumors. / Nageswara Rao, Amulya A.; Scafidi, Joseph; Wells, Elizabeth M.; Packer, Roger J.

In: Pediatric Neurology, Vol. 46, No. 4, 01.04.2012, p. 203-211.

Research output: Contribution to journalReview article

Nageswara Rao, Amulya A. ; Scafidi, Joseph ; Wells, Elizabeth M. ; Packer, Roger J. / Biologically targeted therapeutics in pediatric brain tumors. In: Pediatric Neurology. 2012 ; Vol. 46, No. 4. pp. 203-211.
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