Pediatric Oncology In Vivo Testing Program Coordinating Center

PROJECT SUMMARY
The overarching goal of NCI's Pediatric In Vivo Testing Program (Ped-In Vivo-TP) is to improve outcomes of
pediatric cancer patients and to satisfy the requirements of the Research to Accelerate Cures and Equity
(RACE) for Children Act to assess the efficacy of targeted anti-cancer agents developed for adults in pediatric
contexts. To coordinate the activities of the new Ped-In Vivo-TP initiative, we have assembled a team of
investigators from The Jackson Laboratory (JAX) and Seven Bridges Genomics (SB) with unique combined
expertise and experience with in vivo cancer models, scalable cloud-based data management and analysis
systems, informatics resource development, and multi-site project coordination. Combining the complementary
strengths of JAX and SB provides the ideal foundation for a coordinating center to maximize the short- and
long-term impacts of the Pediatric In Vivo Testing Program for advancing the application of precision medicine
in pediatric oncology. We will manage a comprehensive and cohesive testing program to advance precision
medicine in pediatric oncology through effective public-private partnerships among pharmaceutical companies,
regulatory agencies, funders, and research organizations. We will achieve this goal through the following aims:
Aim 1: Establish and maintain the Pediatric In Vivo Testing Coordinating Center (PIVOT CC) to provide
administrative and logistical support for diverse stakeholders in the Pediatric in Vivo Testing Program
consortium. We will draw on our team's combined decades of experience with multi-site program
management, cancer model development, standardized testing of in vivo cancer models, data management
and analysis, and informatics resource development to ensure timely decision making, conformance to
standard protocols, resource tracking, and effective communications within the Ped-In Vivo-TP. Aim 2:
Provide data management, statistical, and bioinformatics support to ensure data security and integrity.
We will leverage existing protocols and software systems at JAX and SB to identify relevant in vivo cancer
models and to collect, analyze, and securely manage data generated from testing centers within the
consortium. We will perform statistical and bioinformatic analyses on consortium data to reliably inform the
evaluation of the efficacy of novel therapeutic agents in a pediatric oncology setting. We will develop a public-
facing data portal for sharing of data and methods with the broader scientific community. Aim 3: Provide
scientific coordination to maintain an efficient and effective preclinical testing pipeline. We will draw
from the extensive experience of our team with in vivo pharmacology and coordination of similar consortia to
manage and coordinate the testing of agents by the Ped-In Vivo-TP centers at all stages of the process, from
the identification of relevant in vivo models to the generation of final technical reports and publication of results.
We will develop, record, and track performance metrics for the consortium to inform the evaluation of the
program's success and impact.

Grant Number: 5U24CA263963-05
NIH Institute/Center: NIH
Principal Investigator: CAROL BULT