Related Experiment Video
Updated: Jul 12, 2026
Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies
Published on: September 30, 2016
The ITCC-P4 PDX Platform Enables Preclinical Testing of Pediatric Cancers
Aniello Federico1, Apurva Gopisetty1, Didier Surdez2
1Hopp Children's Cancer Center Heidelberg Heidelberg Germany.
Abstract:
Cancer is the leading cause of disease-related deaths among children in high-income countries. Tumor heterogeneity and lack of mechanism-of-action-based therapeutic options are key challenges to overcome to improve pediatric cancer patient survival. To address these challenges, we formed the EU-IMI-2 funded public-private partnership "ITCC-Pediatric Preclinical Proof-of-Concept Platform" (ITCC-P4), which built a large repertoire of patient-derived xenograft (PDX) models representing all major high-risk solid pediatric cancer types for in vivo drug testing. A total of 353 PDX models were established from diagnostic and relapsed pediatric cancers and molecularly characterized, together with matched germline/tumor samples. Serial PDX models were also established, spanning diagnostic/posttreatment, primary/relapse, and metastasis-derived pairs. Proof-of-concept in vivo drug screening data in neuroblastoma and rhabdomyosarcoma models identified potential predictive biomarkers for targeted therapy. Molecular data from the PDX models, accessible at https://r2platform.com/itcc-p4, allowed the selection of models for preclinical testing based on oncogenic drivers and/or potential biomarkers. Operated by a non-profit entity, this sustainable platform aids academic and industrial researchers in developing and prioritizing innovative therapies for pediatric cancer.
Insights
A new platform established over 350 patient-derived xenograft models for pediatric cancers. This resource aids researchers in developing and prioritizing novel targeted therapies for improved pediatric cancer survival.
Area of Science:
- Oncology
- Translational Medicine
- Biobanking
Background:
- Pediatric cancers pose significant mortality challenges in high-income nations.
- Tumor heterogeneity and limited targeted therapies hinder survival rates in children with cancer.
- The ITCC-P4 platform was created to address these critical issues in pediatric oncology.
Purpose of the Study:
- To establish a comprehensive platform of patient-derived xenograft (PDX) models for pediatric cancers.
- To facilitate in vivo drug testing and identify predictive biomarkers for targeted therapies.
- To support academic and industrial research for developing innovative pediatric cancer treatments.
Main Methods:
- Creation of a large repertoire of 353 patient-derived xenograft (PDX) models from various pediatric cancers.
- Comprehensive molecular characterization of PDX models and matched patient samples.
- Establishment of serial PDX models representing different disease stages and relapse.
- In vivo drug screening in neuroblastoma and rhabdomyosarcoma models to identify biomarkers.
Main Results:
- Successfully established 353 PDX models covering major high-risk pediatric solid tumors.
- Identified potential predictive biomarkers for targeted therapies through drug screening in specific models.
- Generated serial PDX models to study tumor evolution and treatment resistance.
- Molecular data is publicly accessible via the R2 platform for model selection.
Conclusions:
- The ITCC-P4 platform provides a valuable resource for preclinical pediatric cancer research.
- The platform enables the selection of PDX models based on specific oncogenic drivers and biomarkers.
- Facilitates the development and prioritization of novel therapeutic strategies for pediatric cancer patients.
