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Updated: Aug 8, 2026

Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
Published on: February 6, 2015
Development of molecular targeted anticancer agents: successes, failures and future directions
1Department of Medical Oncology and Hematology, Princess Margaret Hospital, 610 University Avenue, Toronto, Ontario M5G 2M9, Canada. eric.chen@uhn.on.ca
Abstract:
Recent advances in molecular biology have identified numerous steps and proteins involved in malignant transformation as targets of anticancer therapy. Many molecular targeted agents are now undergoing clinical development. Successful developments of trastuzumab in treating breast cancer, imatinib in chronic myeloid leukemia (CML) and gastrointestinal stromal tumors (GISTs), and bevacizumab in colorectal cancer, have validated the concept of molecular targeting and raised expectations of patients and oncologists alike. Despite these successes, many agents, notably matrix metalloproteinase inhibitors (MMPIs), have failed in their development. In this review, we will address several issues related to tumor biology and clinical trial design that might have contributed to these successes and failures, and discuss strategies to best optimize the development of these novel agents.
Insights
Molecular targeted therapy shows promise in cancer treatment, with successes like trastuzumab and imatinib. However, challenges in tumor biology and clinical trial design impact the development of novel anticancer agents.
Area of Science:
- Molecular biology
- Oncology
- Drug development
Background:
- Malignant transformation involves numerous molecular steps and proteins targeted by anticancer therapies.
- Several molecular targeted agents are in clinical development, building on successes like trastuzumab, imatinib, and bevacizumab.
- Despite validated successes, some agents, such as matrix metalloproteinase inhibitors, have faced developmental failures.
Purpose of the Study:
- To review factors contributing to the success and failure of molecular targeted agents in cancer therapy.
- To discuss challenges in tumor biology and clinical trial design affecting drug development.
- To propose strategies for optimizing the development of novel anticancer agents.
Main Methods:
- Literature review of molecular targeted agents in oncology.
- Analysis of clinical trial outcomes for targeted therapies.
- Discussion of tumor biology and clinical trial design considerations.
Main Results:
- Trastuzumab, imatinib, and bevacizumab exemplify successful molecular targeting in cancer.
- Matrix metalloproteinase inhibitors represent a class of agents that have encountered developmental hurdles.
- Tumor biology complexity and suboptimal clinical trial design can impede therapeutic progress.
Conclusions:
- Molecular targeting has proven effective in specific cancers, validating the approach.
- Further optimization of development strategies is crucial for novel anticancer agents.
- Addressing tumor biology intricacies and refining clinical trial designs are key to future success.
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