# Washington University PDX Development and Trial Center

> **NIH NIH U54** · WASHINGTON UNIVERSITY · 2021 · $1,252,124

## Abstract

With the discovery of a number of new molecular targets and exponential increase in the number of anti-cancer
agents, it has become imperative to optimize preclinical models in order to design rational clinical trials.
Recognizing the unique advantage of the patient-derived xenografts (PDXs) models in drug testing and
personalized medicine, physicians and researchers at Washington University School of Medicine (WUSM) and
the Siteman Cancer Center have been actively generating and utilizing PDX models for cancer research and
the evaluation of molecularly targeted agents. Building on our strength and existing infrastructure, we propose
to establish the Washington University PDX Development and Trial Center (WU-PDTC), as part of PDXNet
program, to promote preclinical testing in a collaborative nation-wide effort.
The PDX core within WU-PDTC will develop and characterize at least 1000 new pathogen-free PDX models
across major tumor types. The PDX models will be genomically and phenotypically characterized using the
latest ‘omics technologies and expertly analyzed using the most current data analysis pipelines that have been
deployed for other large scale NCI programs. The Bioinformatics Core will integrate these analyzes with clinical
annotation from the originating patient to include patient treatment history and tumor response (Aim 1). Our two
research projects will conduct PDX clinical trials using single agent and combinational agents using drugs
under NCI-IND (Aim 2). Project 1 will test pan- or beta isoform specific class I PI3K inhibitors in over 100
breast PDX models while Project 2 will study combinatorial approaches that overcome tumor intrinsic and
extrinsic mechanisms to ERK inhibition in over 100 already available pancreatic PDX models. Proteogenomic
and clinical response data will be collected in these models, as part of a broader effort of characterizing PDX
models and conducting clinical correlation and treatment response analyses by the Bioinformatics Core (Aim
3). All relevant information, including proteogenomic features of PDX models and treatment/response history,
will be tracked in a dedicated relational database that will be accessible to PDXNet and PDMR-FNLCR. The
goal is to identify candidates for human clinical trials for the ET-CTN. In Aim 4, the WU-PDTC will leverage
existing expertise and programs from the NCI-designated Comprehensive Siteman Cancer Center, Institute of
Clinical and Translational Research (ITCS), McDonnell Genome Institute, Mallinkrodt Imaging Research
Center, and Early Therapeutic Clinical Trials Network (ET-CTN) to support the goals of developing and utilizing
PDX models to test and improve cancer treatment, in collaboration with other components of the PDXNet.
Finally, WU-PDTC, through coordination by the Administration Core, will support pilot research projects
utilizing the PDX resources and fostering collaboration across PDTCs, PNMR-FNLCR, and other NCI
programs. The goal is to increase the spectrum...

## Key facts

- **NIH application ID:** 10246866
- **Project number:** 5U54CA224083-04
- **Recipient organization:** WASHINGTON UNIVERSITY
- **Principal Investigator:** Li Ding
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $1,252,124
- **Award type:** 5
- **Project period:** 2017-09-30 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10246866

## Citation

> US National Institutes of Health, RePORTER application 10246866, Washington University PDX Development and Trial Center (5U54CA224083-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10246866. Licensed CC0.

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