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Updated: May 21, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Proof of concept to clinical confirmation: evolving clinical trial designs for targeted agents
1Hematology/Oncology, Mayo Clinic Florida, Jacksonville, FL 32224, USA.
Abstract:
No single therapy benefits the majority of patients in the practice of oncology as responses differ even among patients with similar tumor types. The variety of response to therapy witnessed while treating our patients supports the concept of personalized medicine using patients' genomic and biologic information and their clinical characteristics to make informed decisions about their treatment. Personalized medicine relies on identification and confirmation of biologic targets and development of agents to target them. These targeted agents tend to focus on subsets of patients and provide improved clinical outcomes. The continued success of personalized medicine will depend on the expedited development of new agents from proof of concept to confirmation of clinical efficacy.
Insights
Personalized medicine in oncology uses patient genomic data to tailor treatments, improving outcomes. Continued success requires faster development of targeted therapies for specific patient groups.
Area of Science:
- Oncology
- Genomics
- Personalized Medicine
Background:
- Cancer treatment responses vary significantly among patients, even with similar tumor types.
- The practice of oncology highlights the need for individualized treatment strategies.
Purpose of the Study:
- To support the concept of personalized medicine in oncology.
- To emphasize the use of genomic and biologic information for informed treatment decisions.
Main Methods:
- Utilizing patients' genomic and biologic information.
- Integrating clinical characteristics for treatment planning.
- Identifying and confirming biologic targets for drug development.
Main Results:
- Personalized medicine approaches focus on specific patient subsets.
- Targeted agents demonstrate potential for improved clinical outcomes.
Conclusions:
- Personalized medicine offers a pathway to optimize cancer treatment.
- Expedited development of novel targeted agents is crucial for future success.
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