Precision Medicine Advances for Children With Neuroblastoma


Next Generation Personalized Neuroblastoma Therapy (NEPENTHE) Gives Hope to Children with Relapse or Refractory Disease


November 15, 2016 – NEW YORK, NY: An innovative clinical trial is now enrolling pediatric cancer patients with neuroblastoma shown resistance to traditional treatment protocols. This Next Generation Personalized Neuroblastoma Therapy – also known as NEPENTHE (Greek for “medicine for sorrow”) – is the first precision medicine trial for children that will robustly analyze the genomics of their cancer and use combinations of investigational drugs to target specific mutations in their tumors.

Precision medicine allows cancer researchers to identify genetic abnormalities within a tumor type and to try to match drugs that may interfere with tumor growth accordingly. Although researchers have discovered few specific genetic abnormalities for such targeting among children diagnosed with neuroblastoma, there is now an improved understanding of neuroblastoma biology, which has rationally designed therapies with new potentially effective drugs that may produce dramatic responses in children with the chemo-resistant disease, giving tremendous hope to families who are desperately seeking new treatment options.

Solving Kids’ Cancer (SKC) together with Wade’s Army announce their joint financial support of this Phase 1 clinical trial to test the safety and efficacy of various investigational drugs that will leverage precision medicine for children with neuroblastoma.

NEPENTHE is now open for enrolling children with relapse or refractory neuroblastoma. The trial is being led by Dr. Yael Mosse, Children’s Hospital of Philadelphia (CHOP) and allows children ages 1 year to 21 years to participate.

“We believe precision medicine may provide hope for children with relapse or refractory neuroblastoma, whose prognosis has historically been extremely poor,” said Dr. Mosse. “With this sophisticated and adaptive new trial design, we can offer neuroblastoma patients a ‘matched’ treatment protocol and add new drugs as they become available. We are confident that it will enable us to learn which combinations of investigational drugs will result in significant anti-cancer activity.”

Existing chemotherapy protocols cure less than half of all high-risk neuroblastoma cases. Genetic sequencing coupled with precision medicine shows promise for a strong response to targeted treatment, with fewer long-term toxicities in children.

This study will be done in two parts. First, the tumor will be accessed via biopsy at the time of relapse and subjected to deep sequencing to identify protocol-specified biomarkers for therapy assignment. Then, if the tumor contains a genetic change defined as being actionable, participants will be assigned to therapy based upon the genetic changes identified in the tumor biopsy.

“We are excited to be involved in this ground-breaking precision medicine trial for children with neuroblastoma,” added Scott Kennedy, Executive Director of Solving Kids’ Cancer. “Our partnership with like-minded nonprofits enables us to further our mission to support the development of new pediatric treatment options, such as the NEPENTHE trial, and we are incredibly hopeful about the potential outcome.”



Solving Kids’ Cancer (SKC) focuses on aggressive childhood cancers with low survival rates – Because Every Kid Deserves to Grow Up. SKC helps accelerate new, next-generation treatments, including immunotherapy, cancer vaccines, and new drugs by applying an understanding of the entire childhood cancer research landscape to wisely invest in innovative projects. Visit, @SolveKidsCancer, or for more information.


Wade’s Army’s objective is to bring the fight against neuroblastoma in honor of Wade DeBruin and his battle against the disease. Wade’s Army has a goal: Fund research and supports the children and families affected by their often-fatal form of pediatric cancer. Visit, @WadesArmyHQ, or for more information.

Leave a Comment