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Updated: Jun 11, 2025

Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies
Published on: September 30, 2016
Precision preclinical modeling to advance cancer treatment
David H Gutmann1, Jesse S Boehm2,3, Elinor K Karlsson2,4
1Department of Neurology, Washington University, St Louis, MO 63110, United States.
Abstract:
A new era of cancer management is underway in which treatments are being developed for the entire continuum of the disease process. The availability of genetically engineered and naturally occurring preclinical models serves as instructive platforms for evaluating therapeutic mechanisms. However, a major clinical challenge is that the entire malignancy process occurs across multiple scales including genetic mutations, malignant changes in cell behavior, dysregulated tumor microenvironments, and systemic adaptations in the host. A multidisciplinary group of investigators coalesced at the National Cancer Institute Oncology Models Forum with the overall goal to provide updates on the use of precision preclinical models of cancer. The benefits and limitations of preclinical models were discussed to identify strategies for maximizing opportunities in modeling that could inform future cancer prevention and treatment approaches. Our shared perspective is that the continuum of single cell, multicell, organoid, and in situ models are remarkable resources for the clinical challenges ahead. We provide a roadmap for parsing already available models and include preliminary recommendations for the application of next-generation preclinical modeling in cancer intervention.
Insights
Precision preclinical cancer models are crucial for understanding and treating the disease across multiple scales. This research outlines strategies for utilizing these models to advance cancer prevention and therapy.
Area of Science:
- Oncology
- Translational Cancer Research
- Preclinical Modeling
Background:
- Cancer treatment is evolving to address the entire disease continuum.
- Preclinical models are essential for evaluating therapeutic mechanisms but face challenges in representing multi-scale malignancy.
- The National Cancer Institute Oncology Models Forum convened to discuss precision preclinical models.
Purpose of the Study:
- To provide updates on the application of precision preclinical cancer models.
- To discuss the benefits and limitations of existing preclinical models.
- To identify strategies for optimizing preclinical modeling to inform cancer prevention and treatment.
Main Methods:
- Discussion and consensus-building among a multidisciplinary group of investigators.
- Review of the continuum of preclinical models, including single cell, multicell, organoid, and in situ.
- Development of a roadmap for model selection and preliminary recommendations for next-generation modeling.
Main Results:
- Preclinical models offer valuable platforms for studying cancer across genetic, cellular, microenvironmental, and systemic scales.
- A range of models, from single cell to in situ, are available and beneficial for clinical challenges.
- Strategies for maximizing the utility of preclinical models were identified.
Conclusions:
- The continuum of preclinical models represents a significant resource for addressing complex clinical challenges in cancer.
- A roadmap and recommendations are provided for applying next-generation preclinical models in cancer intervention.
- Optimized use of precision preclinical models can significantly inform future cancer prevention and treatment strategies.

