# Core 3: Preclinical Models and  Therapeutics Core

> **NIH NIH P01** · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · 2020 · $206,420

## Abstract

SUMMARY – PATHOLOGY CORE
The Pathology Core (PC) will assemble and characterize the reagents critical for this proposal including the
wide array of engineered mouse models, clinically and molecularly annotated primary samples, patient derived
tumor xenograft (PDTX) models as well as patient derived organoids (PDOs). These resources will be provided
to project leaders to cross validate experimental findings, define the pathogenetic role of defined lesions and
identify their relevance in human tissue samples using PDTX and PDO models. We anticipate that the services
of the PC core will provide the basis for the biological dissection of the genetic lesions described in all three
Projects, leading to a deeper knowledge of how 3D genome organization plays a crucial role in gene
regulation, oncogenic transcriptional programs and the design of more efficient therapeutic approaches.
Translational cancer research has been greatly facilitated by the demonstration that defined genomic defects
are associated with unique functional fingerprints which can be effectively validated in vitro using primary 2D
and 3D cultures and by employing innovative transgenic mouse models and tumor xenografts in
immunocompromised mice. Importantly, primary 2D and 3D cultures and tumor xenografts have been tested
showing that they largely mimic the original patient tumor, including tumor heterogeneity and can effectively
predict response to therapies. Of note, models from primary patient derived cultures or PDTX have proven to
be more informative than conventional cells lines allowing precise determinations and more reliable
assessments. Lastly, as many as 85% of drugs with in vitro activity in established cell lines have failed in
human studies, primarily because of a lack of efficacy in complex systems or in human settings. Thus, our
models are particularly useful for in vivo mechanistic studies (Projects 1-3), and the information they provide
will be instrumental in understanding the pathogenic potential of the defects investigated in this application,
leading to potential targeted therapies (Projects 2 and 3).

## Key facts

- **NIH application ID:** 9952350
- **Project number:** 5P01CA229086-02
- **Recipient organization:** NEW YORK UNIVERSITY SCHOOL OF MEDICINE
- **Principal Investigator:** Giorgio Inghirami
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $206,420
- **Award type:** 5
- **Project period:** — → —

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9952350

## Citation

> US National Institutes of Health, RePORTER application 9952350, Core 3: Preclinical Models and  Therapeutics Core (5P01CA229086-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9952350. Licensed CC0.

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