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Updated: Jun 5, 2025

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Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
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PDX models for functional precision oncology and discovery science
Zannel Blanchard1, Elisabeth A Brown1, Arevik Ghazaryan1
1Department of Oncological Sciences, University of Utah, Huntsman Cancer Institute, Salt Lake City, UT, USA.
Nature Reviews. Cancer
|December 16, 2024
Summary
Patient-derived xenograft (PDX) models are crucial for precision oncology, enabling personalized cancer therapy validation. Functional precision oncology (FPO) enhances these models for more accurate, in vivo and ex vivo treatment prediction.
Area of Science:
- Oncology
- Genomics
- Translational Medicine
Background:
- Precision oncology optimizes cancer treatment by analyzing tumor molecular profiles.
- Patient-derived xenograft (PDX) models are vital for preclinical validation of targeted therapies.
- Functional precision oncology (FPO) integrates functional assays with molecular data for improved patient biology recapitulation.
Purpose of the Study:
- To review advances and limitations of PDX models in precision oncology and FPO.
- To explore the future integration of AI with PDX models for enhanced cancer treatment prediction.
Main Methods:
- Utilizing diverse PDX and PDX-derived models for in vivo and ex vivo functional assays.
- Analyzing genomic landscapes and molecular alterations in tumors.
- Evaluating therapeutic responses based on specific mutations and signaling pathways.
Main Results:
- PDX models effectively recapitulate patient tumor biology for therapy testing.
- FPO assays provide functional insights complementing static molecular measurements.
- Integration of PDX models and FPO shows promise for personalized treatment strategies.
Conclusions:
- PDX and FPO models are essential tools for advancing precision oncology.
- The synergy of AI and PDX models holds potential for revolutionizing cancer treatment prediction.
- Future directions emphasize integrating functional and molecular data for faster, cost-effective, and personalized cancer therapies.

