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Updated: Aug 23, 2026

Protein Target Prediction and Validation of Small Molecule Compound
Published on: February 23, 2024
[Molecular-target drug]
Saburo Sone1, Seiji Yano, Yasuhiko Nishioka
1Department of Internal Medicine and Molecular Therapeutics, Institute of Health Biosiences, The University of Tokushima Graduate School, 3-18-15 Kuramoto-cho, Tokushima 770-8503, Japan.
Abstract:
Cancer metastasis involves the complicated steps of tumor growth, angiogenesis, invasion and adhesion. At present new drugs targeting particular molecule (s) responsible for such cancer progression and metastasis have been developed in clinics. Major endpoints for cancer treatment should be prolongation of survival and maintenance of QOL. However, clinical development of such molecular-target based drugs is associated with difficulties in evaluating the efficacy in phase I/II studies prior to entering phase III study, because many of the targeted drugs seem cytostatic rather than cytocidal to tumors. New approaches incorporating technologies of genomics and proteomics may provide an expanding repertoire of molecular targeted therapeutics for clinical evaluation. In this review, the significance and problems of biomarkers available for clinical evaluation of molecular targeted drugs are discussed.
Insights
Biomarkers are crucial for evaluating new molecular-targeted cancer drugs, which often have cytostatic effects. This review discusses the significance and challenges of using biomarkers in clinical trials for targeted cancer therapeutics.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Context:
- Cancer metastasis involves complex processes like tumor growth, angiogenesis, invasion, and adhesion.
- Molecular-targeted drugs aim to inhibit specific molecules driving cancer progression.
- Clinical evaluation of these drugs faces challenges due to their cytostatic, rather than cytocidal, mechanisms.
Purpose:
- To review the significance of biomarkers in the clinical evaluation of molecular-targeted cancer drugs.
- To discuss the challenges associated with using biomarkers for efficacy assessment in early-phase clinical trials.
- To explore how genomics and proteomics can expand the repertoire of targeted therapeutics.
Summary:
- Molecular-targeted cancer drugs offer new therapeutic avenues but pose challenges in efficacy evaluation.
- Biomarkers are essential for assessing the clinical benefit of cytostatic targeted agents.
- Genomics and proteomics are advancing the development and evaluation of novel targeted therapies.
Impact:
- Improved clinical trial design for molecular-targeted drugs.
- Enhanced ability to predict patient response to targeted therapies.
- Facilitation of personalized medicine approaches in oncology.
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