# Clinical Trials and Translation

> **NIH NIH P01** · CHILDREN'S HOSP OF PHILADELPHIA · 2021 · $335,600

## Abstract

SUMMARY/ABSTRACT 
High-risk neuroblastoma (NB) remains a therapeutic challenge with expected 5-year overall survival of no more 
than 50%, despite advances in the past two decades with myeloablative therapy, differentiating therapy and 
immunologic and genetically targeted therapies. Core B is the translational linchpin of our multi-disciplinary 
and multi-institutional P01, Discovering and Exploiting Mechanisms of Neuroblastoma (NB) Therapy 
Resistance. The goal of Core B will be enabled by the well-established New Approaches to Neuroblastoma 
Therapy (NANT) consortium, and has the overall objective of capitalizing on our clinical trials expertise and 
infrastructure to support the translation of new therapies with demonstrated pre-clinical activity against 
relapsed and refractory (resistant) NB developed in Projects 1-5 into clinical care, in order to ultimately improve 
the poor survival of children with high-risk NB. The three specific aims of this Core are: 1) to provide the 
clinical expertise for translation of preclinical work from Projects 1-5 collaboration with the statistical Core and 
the Project investigators; 2) to provide the established and adaptable infrastructure for rapid implementation 
into early phase clinical trials; 3) provide the infrastructure for embedded correlative studies, and a unique 
biorepository of highly annotated tumor tissues, bone marrow, blood, and radiologic images obtained from 
resistant NB patients, with genomic and pathologic analysis. Core B will obtain safety, mechanistic, and 
response data from phase 1/2 trials to support promising therapies from Projects 1-5 to be tested in larger 
national and international Phase 2 and 3 trials. This highly integrated P01 will allow rapid translation from 
laboratory to patients, with individualized treatment, utilizing more precise biomarkers of response. Thus, 
Core B will contribute substantively to the overall success of the Program and ultimately improved high-risk NB 
patient outcome.

## Key facts

- **NIH application ID:** 10265476
- **Project number:** 5P01CA217959-05
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** JULIE R PARK
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $335,600
- **Award type:** 5
- **Project period:** 2017-09-18 → 2023-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10265476

## Citation

> US National Institutes of Health, RePORTER application 10265476, Clinical Trials and Translation (5P01CA217959-05). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10265476. Licensed CC0.

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