MYCN deregulation as a potential target for novel therapies in rhabdomyosarcoma

Expert Rev Anticancer Ther. 2006 Feb;6(2):217-24. doi: 10.1586/14737140.6.2.217.


Rhabdomyosarcoma is the most common soft-tissue sarcoma of childhood. Treatment requires a multimodality approach combining chemotherapy with surgery and radiotherapy. Although overall outcomes have improved considerably, the outlook for patients with high-risk disease, particularly the alveolar subtype, remains bleak and there is a clear need for new chemotherapeutic strategies. This review focuses on the possibilities for interventions targeting myc myelocytomatosis viral related oncogene, neuroblastoma derived (MYCN). The importance of aberrant expression of this oncogene is well established in neuroblastoma and recent data indicate that MYCN deregulation also occurs in up to a quarter of alveolar subtype cases. A range of possible approaches to target MYCN is discussed, including nucleic acid-based and immunotherapy strategies.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / pharmacology
  • Combined Modality Therapy
  • Gene Expression Regulation, Neoplastic / drug effects*
  • Humans
  • Immunotherapy
  • N-Myc Proto-Oncogene Protein
  • Nuclear Proteins / biosynthesis
  • Nuclear Proteins / genetics*
  • Nucleic Acids / therapeutic use
  • Oligonucleotides, Antisense / therapeutic use
  • Oncogene Proteins / biosynthesis
  • Oncogene Proteins / genetics*
  • Rhabdomyosarcoma / drug therapy*
  • Rhabdomyosarcoma / genetics*
  • Rhabdomyosarcoma / pathology


  • Antineoplastic Agents
  • MYCN protein, human
  • N-Myc Proto-Oncogene Protein
  • Nuclear Proteins
  • Nucleic Acids
  • Oligonucleotides, Antisense
  • Oncogene Proteins