# Pediatric Preclinical In Vivo Testing Center for Pediatric Sarcoma and Other Solid Tumors

> **NIH NIH U01** · SLOAN-KETTERING INST CAN RESEARCH · 2021 · $749,282

## Abstract

ABSTRACT
With recent enactment of the Research to Accelerate Cures and Equity (RACE) for Children Act, there is a
growing impetus to identify pediatric indications for molecularly targeted drugs. Accordingly, there is a pressing
need for clinically relevant preclinical studies that can help prioritize pediatric indications for clinical application
of nearly the entire universe of cancer drugs currently in development. To meet this need, we established a
preclinical testing program that has created >300 genomically-characterized pediatric solid tumor patient-
derived xenograft (PDX) models between the pediatric oncology programs at Memorial Sloan Kettering Cancer
Center and University of California San Francisco. We propose to leverage this large portfolio of models across
a diversity of diseases, along with the deep expertise of the team, to establish a NCI Pediatric In Vivo Testing
Program (Ped-In Vivo-TP) Research Team focused on pediatric bone and soft tissue sarcomas, renal tumors,
desmoplastic small round cell tumor (DSRCT) and other rare pediatric solid tumors. The Aims of this Research
Team are: Aim 1. Integrate with the Coordinating Center and other Research Teams to prioritize agents
for preclinical evaluation. We will leverage our team's translational expertise as well as existing connections
to disease committees within cooperative groups to inform and facilitate new agent selection and preclinical
evaluation in appropriate models to prioritize agents to advance into pediatric oncology clinical trials. Aim 2.
Utilize PDX portfolios representative of disease heterogeneity to assess therapeutic agents. We will
utilize the >300 PDX models from MSKCC and UCSF, supplemented with models from the PROXC consortium
where necessary, to assess 8-10 therapeutic agents/year using study designs matched to the therapeutic
question. All preclinical drug testing will be conducted at MSKCC. Aim 3. Align central and local data
analyses to ensure rigor in results reporting. For the purposes of prioritization, we will maintain equipoise in
validating (“go”) or invalidating (“no go”) therapeutic hypotheses, and will complement central data analyses
with advanced local biostatistical expertise. Aim 4. Translational biomarker discovery. We will leverage the
combined expertise of our Research Team to identify clinically translatable biomarkers predictive of enhanced
response or drug resistance. In some cases, predictive biomarkers may not be genetic, but instead will depend
on transcriptional or protein-based assays of target activity. In other cases, genomically identified biomarkers
have to be translated to clinically utilizable assays. Together, the expertise and capabilities of this Research
Team will support the rigorous evaluation of novel therapeutic hypotheses in clinically-representative models to
enable prioritization and translation of the most promising emerging agents into biomarker-informed clinical
trials for children with high risk solid tumors...

## Key facts

- **NIH application ID:** 10300341
- **Project number:** 1U01CA263967-01
- **Recipient organization:** SLOAN-KETTERING INST CAN RESEARCH
- **Principal Investigator:** Filemon S Dela Cruz
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $749,282
- **Award type:** 1
- **Project period:** 2021-07-01 → 2026-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10300341

## Citation

> US National Institutes of Health, RePORTER application 10300341, Pediatric Preclinical In Vivo Testing Center for Pediatric Sarcoma and Other Solid Tumors (1U01CA263967-01). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10300341. Licensed CC0.

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