# Patient-derived orthotopic xenograft models for cancer disparity studies of pediatric brain tumors

> **NIH NIH U01** · LURIE CHILDREN'S HOSPITAL OF CHICAGO · 2024 · $248,237

## Abstract

This application is prepared in response to the FOA: Administrative Supplements to Support Cancer Disparity
Collaborative Research (PAR-22-114) to support our UO1 grant (2UO1 CA199288-06, active till 06/2026) in the
NIH/NCI Pediatric Preclinical In Vivo Testing Program (PIVOT). Our objective is to characterize the racial/ethnic
nature of a panel of 165 patient-derived orthotopic xenograft (orthotopic PDX or PDOX) models of pediatric brain
tumors, to determine disparities in therapeutic responses among racial/ethnic groups, and to understand genetic
and epigenetic variations that contribute to such disparities. Our long-term goal is to include well-defined animal
model system of diverse racial/ethnic groups to advance cancer disparity research in pediatric brain tumors.
 Brain tumor is the number one cause of cancer related death in children. Similar to many adult cancers,
health disparities have been identified. In addition to socioeconomic and socio-environmental factors, differences
of tumor biology may also play a role, as adult cancers have shown distinct gene expression, DNA methylation
and mutation profiles among racial/ethnic groups. Our hypothesis that the racial/ethnic-specific genetic
components contribute to cancer disparity of pediatric brain tumors and affect their responses to therapy. We
hypothesize that the inclusion of minority patient-derived PDOX models will further support the evaluation and
prioritization of racial/ethnic appropriate anti-cancer drugs. To test these hypotheses, we propose three Specific
Aims. Aim 1: Perform genetic ancestry analysis of our PDOX models to define their racial/ethnic origin.
We will supplement self-reported racial/ethnic information with genetic ancestry analysis with EthSEQ analysis
of RNAseq data and Infinium Global Screening Array (GCD) analysis to our existing PDOX models (including
the 101 models with self-reported racial/ethnic information) to achieve a complete and genetic-based
determination/inferrence of race/ethnicity of our model system. Aim 2: Determine the landscape of active
drugs against group 3 medulloblastoma in Black, Hispanic and White Children. We have 62 models of
medulloblastoma, including 43 models with self-reported race/ethnicity. Since group 3 is the most aggressive
subtype, we will a) complete a high throughput drug screening (8,000 drugs) in 10 models from Black, 10 from
Hispanic and 10 from White children, respectively; b) understand racial/ethnic-specific mechanisms of action by
correlating active drugs with the underlying genetic and epigenetic abnormalities. Aim 3. Integrate minority
derived PDOX models in future preclinical drug testing in NIH/NCI PIVOT program. We will add racial/ethnic
information as a new criteria of model selection to include minority derived PDOX models as often as possible.
Completion of this study will provide a large panel of animal models representing a diverse range of racial/ethnic
groups that can be widely distributed to facilitate bi...

## Key facts

- **NIH application ID:** 10923533
- **Project number:** 3U01CA199288-09S1
- **Recipient organization:** LURIE CHILDREN'S HOSPITAL OF CHICAGO
- **Principal Investigator:** Xiaonan Li
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $248,237
- **Award type:** 3
- **Project period:** 2015-07-14 → 2026-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10923533

## Citation

> US National Institutes of Health, RePORTER application 10923533, Patient-derived orthotopic xenograft models for cancer disparity studies of pediatric brain tumors (3U01CA199288-09S1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10923533. Licensed CC0.

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