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Updated: Mar 10, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Animal testing is still the best way to find new treatments for patients
Silvio Garattini1, Giuliano Grignaschi1
1IRCCS Istituto di Ricerche Farmacologiche Mario Negri, Milan, Italy.
Abstract:
Experimental research proceeds by hypotheses formulated on the basis of previous or new knowledge and then tested. If they are accepted, they serve as the basis for further hypotheses, and if they are rejected new hypotheses can be developed. In other words, when we are at the frontiers of knowledge the path is forged by "trial and error". When a trial shows a hypothesis is wrong, this is a step toward making fewer errors. This process also applies to drug development. There is no magic formula at present to predict - at the pre-clinical level - the therapeutic value of a drug for people with a disease. However, pre-clinical studies are needed in order to formulate hypotheses that justify clinical trials. Without these preliminary studies in vitro and in vivo in selected animal species it would be unethical to test still unproven chemicals in humans.
Insights
Scientific advancement, including drug development, relies on a "trial and error" process. Pre-clinical studies are crucial for forming hypotheses that ethically guide human clinical trials.
Area of Science:
- Pharmacology
- Drug Discovery
- Scientific Methodology
Background:
- Scientific progress advances through hypothesis testing and refinement.
- The path to new knowledge often involves iterative 'trial and error' processes.
- Drug development currently lacks a predictive formula for human therapeutic efficacy at the pre-clinical stage.
Purpose of the Study:
- To underscore the indispensable role of pre-clinical research in drug development.
- To highlight the ethical necessity of hypothesis-driven clinical trials.
- To explain the 'trial and error' methodology in scientific research.
Main Methods:
- Formulating hypotheses based on existing or new knowledge.
- Testing hypotheses through experimental research.
- Utilizing in vitro and in vivo pre-clinical studies in animal models.
Main Results:
- Accepted hypotheses form the basis for further investigation.
- Rejected hypotheses guide the development of new research directions.
- Pre-clinical studies generate essential data to justify human clinical trials.
Conclusions:
- The 'trial and error' method is fundamental to expanding scientific frontiers.
- Pre-clinical research is a non-negotiable prerequisite for ethical human testing.
- Robust pre-clinical data are vital for formulating sound hypotheses in drug development.
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