# NCI Pediatric In Vivo Testing Program: Neuroblastoma

> **NIH NIH U01** · CHILDREN'S HOSP OF PHILADELPHIA · 2024 · $712,799

## Abstract

Project Summary
 Children with disseminated neuroblastoma have a very high risk of treatment failure and death despite
receiving intensified chemotherapy, radiation therapy and immunotherapy. The long-term goal of the Mossé
and Maris translational research programs is to substantively improve neuroblastoma cure rates by developing
patient-specific therapies that target the unique oncogenic drivers of each case. Within the context of the
National Cancer Institute’s Pediatric In Vivo Testing Program (Ped-In Vivo-TP) we propose a Neuroblastoma
Research Program built on richly annotated and highly characterized patient derived xenograft (PDX) and
other recently developed murine models of this disease. The central hypothesis to be tested in this Program is
that neuroblastoma-specific oncogenic drivers and optimal immunotherapeutic targets can be defined and
exploited through rationally designed therapies based on validated and clinically measurable biomarkers.
Through our dedicated focus on neuroblastoma and our central role in the former Pediatric Preclinical Testing
Program and Consortium, we have developed an investigative team and rich set of resources and reagents to
be uniquely positioned to achieve the goals of the Ped-In Vivo-TP. Here we propose to use a large (and
growing) collection of PDX models that have been fully characterized with the most modern genomic
technologies to address the challenge of prioritizing the large armamentarium of anti-cancer agents in
development so that early phase biomarker-driven clinical trials can be designed with the objective of showing
potent and specific anti-tumor activity. We propose three specific aims directed towards 1) developing and
characterizing highly annotated models of human neuroblastoma; 2) performing preclinical trials with drugs
directed against defined therapeutic vulnerabilities in order to prioritize agents for the clinic, and 3) developing
the portfolio of preclinical data required for design of clinical trials with robust biomarkers for patient selection
and monitoring. In collaboration with other preclinical testing programs, we will seek to determine if discoveries
in our program are relevant to other childhood cancers and collaborate across disease groups on clinical
development strategies. Thus, this Program will seek to shift the paradigm for how high-risk neuroblastoma
patients are treated with the goal of substantively improving the outcomes, both in terms of cure rates, but also
by decreasing the toxicity associated with current standards of care.

## Key facts

- **NIH application ID:** 10862829
- **Project number:** 5U01CA263957-04
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** Yael P Mosse
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $712,799
- **Award type:** 5
- **Project period:** 2021-07-01 → 2026-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10862829

## Citation

> US National Institutes of Health, RePORTER application 10862829, NCI Pediatric In Vivo Testing Program: Neuroblastoma (5U01CA263957-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10862829. Licensed CC0.

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