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Updated: May 4, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
[Targeted therapies, prognostic and predictive factors in endocrine oncology]
S Hescot1, E Baudin1, F Borson-Chazot2
1Inserm U693, Faculté de médecine Paris-Sud, Université Paris-Sud, Le Kremlin-Bicêtre, France; Médecine nucléaire et Oncologie endocrinienne, Institut Gustave-Roussy, Université Paris-Sud, Villejuif, France.
Abstract:
A better understanding of molecular mechanisms responsible for tumorigenesis has allowed the development of targeted drugs designed to improve the outcome of cancer. In endocrine tumors, several molecules have demonstrated efficacy in terms of progression free survival during phase III trials such as vandetanib and cabozantinib in medullary thyroid carcinoma, sorafenib in differentiated thyroid carcinoma and everolimus or sunitinib for pancreatic neuroendocrine tumors. Rare cancer network has allowed ongoing phase III trials in malignant pheochromocytoma and adrenocortical carcinoma. However, to date no specific predictive biomarker has yet been identified for a personalized cancer medicine. We review recent advances in endocrine oncology concerning molecular targets identification, targeted therapies and predictive or prognostic markers.
Insights
Targeted therapies show promise in endocrine tumors, improving progression-free survival. However, identifying predictive biomarkers remains crucial for personalized endocrine cancer medicine.
Area of Science:
- Endocrine oncology
- Molecular mechanisms of tumorigenesis
- Targeted cancer therapies
Background:
- Understanding cancer molecular mechanisms aids targeted drug development.
- Targeted therapies like vandetanib, cabozantinib, sorafenib, everolimus, and sunitinib show efficacy in endocrine tumors.
- Ongoing trials investigate treatments for rare endocrine cancers, including malignant pheochromocytoma and adrenocortical carcinoma.
Purpose of the Study:
- To review recent advances in endocrine oncology.
- To discuss molecular targets, targeted therapies, and predictive/prognostic markers.
- To highlight the need for predictive biomarkers in personalized endocrine cancer medicine.
Main Methods:
- Literature review of recent advances in endocrine oncology.
- Analysis of phase III trial data for targeted therapies in endocrine tumors.
- Synthesis of information on molecular targets and biomarkers.
Main Results:
- Several targeted drugs have demonstrated efficacy in improving progression-free survival for specific endocrine tumors.
- Ongoing clinical trials are exploring new treatments for rare endocrine malignancies.
- No specific predictive biomarker has been identified for personalized endocrine cancer treatment yet.
Conclusions:
- Targeted therapies represent a significant advancement in endocrine oncology.
- Further research is needed to identify predictive biomarkers for personalized cancer medicine.
- Personalized treatment strategies are essential for improving outcomes in endocrine tumors.
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