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Serendipity in Cancer Drug Discovery: Rational or Coincidence?
Sahdeo Prasad1, Subash C Gupta2, Bharat B Aggarwal1
1Cytokine Research Laboratory, Department of Experimental Therapeutics, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Abstract:
Novel drug development leading to final approval by the US FDA can cost as much as two billion dollars. Why the cost of novel drug discovery is so expensive is unclear, but high failure rates at the preclinical and clinical stages are major reasons. Although therapies targeting a given cell signaling pathway or a protein have become prominent in drug discovery, such treatments have done little in preventing or treating any disease alone because most chronic diseases have been found to be multigenic. A review of the discovery of numerous drugs currently being used for various diseases including cancer, diabetes, cardiovascular, pulmonary, and autoimmune diseases indicates that serendipity has played a major role in the discovery. In this review we provide evidence that rational drug discovery and targeted therapies have minimal roles in drug discovery, and that serendipity and coincidence have played and continue to play major roles. The primary focus in this review is on cancer-related drug discovery.
Insights
Drug discovery is expensive due to high failure rates. This review suggests serendipity, not rational design, drives most successful novel drug discoveries, especially in cancer research.
Area of Science:
- Pharmacology and Drug Discovery
- Oncology
- Biomedical Research
Background:
- Novel drug development costs up to $2 billion, with high failure rates contributing significantly.
- Targeted therapies for single pathways are insufficient for complex, multigenic diseases.
- Most chronic diseases, including cancer, diabetes, and cardiovascular conditions, are multigenic.
Purpose of the Study:
- To analyze the role of serendipity versus rational design in novel drug discovery.
- To provide evidence supporting the impact of coincidence in identifying successful therapeutics.
- To focus on the implications for cancer-related drug discovery.
Main Methods:
- Review of historical drug discovery processes.
- Analysis of numerous drugs approved for various diseases, including cancer.
- Examination of the discovery pathways for successful therapeutics.
Main Results:
- Serendipity and coincidence have played a major role in the discovery of numerous existing drugs.
- Rational drug discovery and targeted therapies have had minimal impact compared to accidental findings.
- This trend is particularly evident in cancer drug discovery.
Conclusions:
- The discovery of effective drugs relies heavily on chance occurrences rather than purely rational approaches.
- Rethinking drug discovery strategies to incorporate or acknowledge the role of serendipity may be beneficial.
- Future research should investigate how to leverage or identify serendipitous discoveries more effectively, especially in oncology.
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