Childhood Cancer

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Targeting MYCN

Children’s Hospital of Philadelphia
Yael Mossé, MD & Julie Park, MD
Grant Type: 
Crazy 8 Pilots
Year Awarded: 
Type of Childhood Cancer: 
Project Description: 


John M. Maris, MD, Children’s Hospital of Philadelphia 
Martin Eilers, PhD, University of Würzburg
William A. Weiss, MD/PhD, University of California, San Francisco
Frank Speleman, PhD, Ghent University
Steven DuBois, MD, Dana-Farber Cancer Institute
Katherine Matthay, MD, University of California, San Francisco
Michael D. Hogarty, MD, Children’s Hospital of Philadelphia
Garrett M. Brodeur, MD, Children’s Hospital of Philadelphia
Mark A. Lemmon, PhD, Yale School of Medicine
Hubert N. Caron, MD/PhD, Roche

Project Summary

Since the original description in 1983 of MYCN amplification as the first described oncogene in human cancer from studies of neuroblastoma cell lines, an intense focus has been placed on exploiting the possibility of targeting this cancer gene. MYCN and its sister oncogene MYC are ideal drug targets in cancer. MYCN fits all the requirements of such a molecule because it is one of the few proteins that is always upregulated in cancer. The MYC family of genes play crucial roles in a plethora of human cancers, including multiple myeloma and Burkitt lymphoma, neuroblastoma, medulloblastoma, small cell lung cancer and a subset of prostate cancers. The seemingly insurmountable challenge of targeting MYC(N) is now evolving as our knowledge of MYC(N) biology and recent technological advances are providing an exceptional opportunity to reach the goal of targeting MYC(N) tumors. This proposal seeks to explore innovative approaches and utilize cutting-edge technologies for attacking the proteins encoded by MYCN as well as MYCN itself to ultimately create effective therapies for MYCN-addicted cancers.