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Updated: Jul 11, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Molecular-targeted therapies: lessons from years of clinical development
Daniela D Rosa1, Gustavo Ismael, Lissandra Dal Lago
1Medical Oncology Clinic, Jules Bordet Institute, and L Universite Libre de Bruxelles (ULB), Brussels, Belgium. dornellesrosa@hotmail.com
Abstract:
Over the past decade, molecular-targeted therapies have been added to cytotoxic and anti-endocrine drugs in the treatment of cancer, with the aim to target the molecular pathways that underlie the carcinogenic process and maintain the cancer phenotype. Success with some of these agents has suggested that identification and validation of the drug target is the starting point for the route of development of active, safe and effective drugs. Main molecular targets used to the development of anticancer drugs are cell surface receptors, signal transduction pathways, gene transcription targets, ubiquitin-proteasome/heat shock proteins and tumour microenvironment components (especially antiangiogenic agents). Here, we review the development of the main molecular targeted non-cytotoxic agents studied in cancer, highlighting lessons derived from the development of these novel drugs and proposing new horizons for the clinical development of molecular-targeted therapies.
Insights
Molecular-targeted therapies offer new cancer treatment options by targeting specific pathways. This review highlights key targets and lessons learned for developing effective, safe anticancer drugs.
Area of Science:
- Oncology
- Pharmacology
- Molecular Biology
Background:
- Molecular-targeted therapies complement traditional cytotoxic and anti-endocrine treatments for cancer.
- These therapies aim to disrupt specific molecular pathways driving cancer growth and survival.
- Successful targeted agents underscore the importance of target identification and validation.
Purpose of the Study:
- To review the development of major molecular-targeted, non-cytotoxic anticancer agents.
- To highlight key lessons learned from the clinical development of these novel therapies.
- To propose future directions for molecular-targeted drug development in oncology.
Main Methods:
- Review of scientific literature on molecular-targeted therapies in cancer treatment.
- Analysis of main molecular targets utilized in anticancer drug development.
- Examination of clinical development pathways and outcomes for targeted agents.
Main Results:
- Key molecular targets include cell surface receptors, signal transduction pathways, gene transcription, ubiquitin-proteasome/heat shock proteins, and tumor microenvironment components.
- Antiangiogenic agents represent a significant component of targeted therapies.
- Development of targeted therapies has yielded valuable insights into drug efficacy and safety.
Conclusions:
- Molecular-targeted therapies represent a significant advancement in cancer treatment.
- Continued focus on target validation and understanding cancer biology is crucial for future drug development.
- Exploring new horizons in clinical development will enhance the effectiveness of targeted anticancer strategies.
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