Childhood Cancer

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Drugging MYCN

Children's Hospital of Philadelphia
Yael Mossé, MD
Grant Type: 
Crazy 8 Awards
Year Awarded: 
Type of Childhood Cancer: 
Brain Tumors, Medulloblastoma, Neuroblastoma
Project Description: 

Project Team
John Maris, MD, Children's Hospital of Philadelphia
Ophir Shalem, PhD, Children's Hospital of Philadelphia
Martin Eilers, PhD, University of Würzburg
Elmar Wolf, PhD, University of Würzburg
Martine F. Roussel, PhD, St. Jude Children's Research Hospital
Natalia Jura, PhD, University of California San Francisco
William A. Weiss, MD/PhD, University of California San Francisco
Kliment A. Verba, PhD, University of California San Francisco
Seychelle Vos, PhD, MIT
Gwenn Hansen, PhD, Nurix Therapeutics

Childhood cancers are caused by genetic mutations that lead to changes in proteins that are essential for normal human development. The protein MYCN is the cause of many of the most aggressive pediatric cancers, including high-risk neuroblastoma and medulloblastoma. One of the holy grails of pediatric cancer drug development is finding a way to directly target MYCN in patient tumors, but this has been elusive. To address this major unmet need, we have developed an international team of scientists with complementary expertise to attack this problem with innovative new technologies. In this project, we plan to “drug the undruggable” by developing hybrid molecules that can specifically attach to MYCN and also then trick the cancer cells own internal machinery to dissolve MYCN, which will lead to cancer cell death. We will develop these drugs with Nurix Therapeutics and their innovative drug development platform, and our research will be overseen by a Scientific Advisory Board of the top MYCN and drug development experts.

Project Goal
The goal is to develop such a MYCN-direct drug in four years, prove that it is effective against the most lethal pediatric cancers in our collaborative laboratories, and be ready to launch a clinical trial shortly after the grant is completed. Successful completion of this project as planned will lead to curative new drugs for children with currently incurable cancers.

Co-funded by: 
Kate's Cause
Sammy’s Superheroes Foundation