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Next-Generation Personalized Neuroblastoma Therapy (Nepenthe)

Children's Hospital of Philadelphia
Yael Mosse, MD & John Maris, MD
Grant Type: 
Bio-Therapeutic Impact Award
Year Awarded: 
Type of Childhood Cancer: 
Project Description: 

Project Update (July 2019)

The clinical trial for Next Generation Personalized Neuroblastoma Therapy (NEPENTHE) is now enrolling patients. The purpose of this study is to match genomic aberrations in tumor cells at time of relapse to rationally designed combinations of molecularly targeted agents. This study will be done in two parts:

  • Part I: Tumor will be accessed at study entry via a biopsy and subjected to deep sequencing to identify protocol-specified biomarkers for therapy assignment. 
  • Part II: If the tumor contains a genetic change defined by the study as being actionable, and other criteria are met, participants will be assigned to therapy based upon the genetic changes identified in the tumor biopsy.

ContactAlana Fitzsimmons

Full details of the trial.


Neuroblastoma remains a devastating clinical problem and continues to be a leading cause of childhood cancer morbidity and mortality despite dramatic increases in therapy. Recent developments in cancer research are changing our understanding of neuroblastoma biology and treatment on the basis of the molecular underpinnings of individual tumors, and are challenging us to design more rational and less toxic therapies.

Project Goal

We propose a paradigm-shifting collaborative clinical trial for children with relapsed or refractory neuroblastoma to identify the genetic features of the cancer at time of relapse that predict drug efficacy, and to provide combination therapies to target these vulnerabilities. We have designed a program to allow for the addition of new therapies based on ongoing laboratory studies, with the overarching goal of substantially changing how we think about neuroblastoma at relapse and how these insights may inform the design of upfront clinical trials for newly diagnosed patients with high-risk neuroblastoma.

Co-funded by: 
Northwestern Mutual Foundation