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Published on: February 6, 2015
Assessing intraepithelial neoplasia and drug safety in cancer-preventive drug development
Gary J Kelloff1, Caroline C Sigman
1National Institutes of Health, National Cancer Institute, Division of Cancer Treatment and Diagnosis, Executive Plaza North Room 6058, 6130 Executive Boulevard, Rockville, Maryland 20852, USA. kelloffg@mail.nih.gov
Abstract:
Despite significant interest from the research community and the population in general, drug approvals for cancer prevention and/or cancer risk reduction are few. This is due, in part, to the requirement that new cancer-preventive drugs must first be shown to be efficacious in reducing cancer incidence or mortality. Moreover, such drugs need to have proven safety for long-term administration. This process can be improved by focusing on precancer (intraepithelial neoplasia) to identify subjects at risk and prove efficacy in shorter, smaller trials as well as on detecting early markers of potential toxicities of chronic exposure to cancer-preventive drug regimens.
Insights
Drug approvals for cancer prevention are limited. Focusing on precancerous conditions and early toxicity markers can accelerate the development of safe and effective cancer-preventive drugs.
Area of Science:
- Oncology
- Pharmacology
- Preventive Medicine
Background:
- Few drugs are approved for cancer prevention or risk reduction.
- Drug development requires demonstrating efficacy in reducing cancer incidence or mortality and proven long-term safety.
Purpose of the Study:
- To improve the drug approval process for cancer prevention.
- To identify strategies for more efficient and effective drug development in cancer prevention.
Main Methods:
- Focusing on precancerous conditions (intraepithelial neoplasia) to identify at-risk individuals.
- Utilizing early markers for potential toxicities associated with long-term drug exposure.
Main Results:
- Suggests that targeting precancerous lesions can shorten trial durations and reduce sample sizes.
- Highlights the importance of early toxicity detection for long-term safety assessment.
Conclusions:
- The current drug approval pathway for cancer prevention is lengthy and demanding.
- Shifting focus to precancerous conditions and early toxicity markers offers a viable strategy to expedite the development of cancer-preventive drugs.
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