Review

Advances in Therapy

, Volume 29, Issue 11, pp 925-934

First online:

Cabozantinib for the Treatment of Advanced Medullary Thyroid Cancer

  • Madhavi NagillaAffiliated withSection of Endocrinology, Diabetes and Metabolism, Department of Medicine, University of Chicago
  • , Rebecca L. BrownAffiliated withSection of Endocrinology, Diabetes and Metabolism, Department of Medicine, University of Chicago Email author 
  • , Ezra E. W. CohenAffiliated withSection of Endocrinology, Diabetes and Metabolism, Department of Medicine, University of Chicago

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Abstract

Introduction

Patients with advanced medullary thyroid cancer (MTC) have poor prognoses and limited treatment options. Improved knowledge about molecular aberrations associated with MTC and the availability of novel targeted tyrosine kinase inhibitors (TKIs) have led to new potential treatment modalities. Cabozantinib is an oral multitargeted TKI with activity against multiple receptors including RET, vascular endothelial growth factor receptor type 2 (VEGFR2), and MET that has been evaluated in MTC in the preclinical and clinical arenas.

Methods

This article reviews unmet clinical needs in advanced MTC. The authors consider novel agents that have been studied in MTC, with a focus on the investigational agent cabozantinib. Up-to-date clinical data of cabozantinib in MTC are discussed.

Results

Recent clinical evaluation suggests that cabozantinib is the first agent to prolong progression-free survival in patients with progressive MTC. These findings indicate that cabozantinib may be an effective therapy in advanced MTC. No improvement in overall survival has been demonstrated but data are not mature.

Conclusion

Cabozantinib may be an effective treatment option for patients with advanced MTC and is worthy of further evaluation.

Keywords

Cabozantinib Endocrinology Medullary thyroid cancer Oncology Targeted therapy Tyrosine kinase inhibitors