Editorial: Targeting MYCN in pediatric cancers

Abstract

MYCN is the product of a gene frequently deregulated in childhood tumors that belongs to a small but very famous family of transcription factors whose prototype member is c-MYC. The other member of the family is L-MYC, identified as a gene amplified in a subset of lung cancers. c-MYC is widely expressed in normal tissues, is the most deregulated protooncogene in human cancer, and not surprisingly, it is the subject of intensive investigations in many laboratories worldwide. Clearly, decoding its function in tumorigenesis and finding ways of inhibiting its oncogenic activity would have a very large impact in terms of human health. In contrast to the broad significance of c-MYC, the expression of MYCN is temporally and spatially restricted during embryonal development, being detected mostly in cells of the developing nervous system. This more limited function is also reflected in human pathology, with only a few types of tumors presenting alterations of MYCN. These cancers arise in the nervous system, both central and peripheral, manifesting as medulloblastomas, gliomas, and neuroblastomas. Despite the fact that MYCN-positive tumors are relatively rare, their very aggressive nature and the pediatric setting make therapeutic treatments a clinical challenge.