Targeted therapy in pediatric and adolescent oncology

Cancer. 2011 May 15;117(10 Suppl):2268-74. doi: 10.1002/cncr.26050.


Cancers in children and adolescents are fortunately infrequent. Overall, cure rates are good, approximately 80%, although this varies by histology and stage. Targeted therapies aim to improve efficacy and decrease toxicity by more specifically affecting malignant cells or their supporting stroma. Cancers of early life are often of different histology than those seen in adults. Sometimes, the same pathway is affected, even if the histology is different. Toxicities may also be different, particularly in younger children. These factors render drug development in young people challenging. This article reviews some successes and challenges to that development, including brief discussions of imatinib, lestaurtinib, antiangiogenesis, and anti-GD2 therapies.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adolescent
  • Angiogenesis Inhibitors / therapeutic use
  • Benzamides
  • Child
  • Gangliosides / antagonists & inhibitors
  • Humans
  • Imatinib Mesylate
  • Molecular Targeted Therapy / methods*
  • Neoplasms / drug therapy*
  • Pharmacokinetics
  • Piperazines / therapeutic use
  • Pyrimidines / therapeutic use
  • Young Adult
  • fms-Like Tyrosine Kinase 3 / antagonists & inhibitors


  • Angiogenesis Inhibitors
  • Benzamides
  • Gangliosides
  • Piperazines
  • Pyrimidines
  • ganglioside, GD2
  • Imatinib Mesylate
  • FLT3 protein, human
  • fms-Like Tyrosine Kinase 3