Neuroblastoma: molecular pathogenesis and therapy

Annu Rev Med. 2015:66:49-63. doi: 10.1146/annurev-med-011514-023121. Epub 2014 Oct 27.


Neuroblastoma is a developmental tumor of young children arising from the embryonic sympathoadrenal lineage of the neural crest. Neuroblastoma is the primary cause of death from pediatric cancer for children between the ages of one and five years and accounts for ∼13% of all pediatric cancer mortality. Its clinical impact and unique biology have made this aggressive malignancy the focus of a large concerted translational research effort. New insights into tumor biology are driving the development of new classification schemas. Novel targeted therapeutic approaches include small-molecule inhibitors as well as epigenetic, noncoding-RNA, and cell-based immunologic therapies. In this review, recent insights regarding the pathogenesis and biology of neuroblastoma are placed in context with the current understanding of tumor biology and tumor/host interactions. Systematic classification of patients coupled with therapeutic advances point to a future of improved clinical outcomes for this biologically distinct and highly aggressive pediatric malignancy.

Keywords: cancer biology; epigenetics; epithelial-to-mesenchymal transition; neural crest development; neuroblastoma; oncogenes; pediatric cancer.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Epithelial-Mesenchymal Transition*
  • Humans
  • Infant
  • Infant, Newborn
  • Neural Crest / embryology*
  • Neuroblastoma / embryology*
  • Neuroblastoma / metabolism
  • Neuroblastoma / therapy