By understanding the genetic features of a tumor, researchers are able to use specific drugs that interfere with certain cell processes that cause unrestricted growth. This is the first precision medicine trial for children that will robustly analyze the genomics and epigenomics of cancer and use combinations of investigational drugs to target specific mutations and alterations in the tumors.

Project Title: NEPENTHE (Match NB) – Phase I Trial Matching Targeted Agents Based on Next-Generation Sequencing
Researcher: Yael Mosse, MD
Institution: Children’s Hospital of Philadelphia
Study Type: Phase I clinical trial
Status:  Ongoing

By understanding the genetic features of a tumor, researchers are able to use specific drugs that interfere with certain cell processes that cause unrestricted growth. Although researchers have discovered a few specific genetic abnormalities for neuroblastoma, a few potentially effective investigational drugs have also been identified that may produce dramatic responses in children with chemo-resistant disease. This is the first precision medicine trial for children that will analyze the genomics and epigenomics of this cancer and use combinations of investigational drugs to target specific mutations and alterations in the tumors.

To learn more about this trial, visit clinicaltrials.gov

Charity Partners: Wade’s Army, Ellen’s Friends and Family