Related Experiment Video
Updated: May 16, 2026

Spontaneous Murine Model of Anaplastic Thyroid Cancer
Published on: February 3, 2023
Vandetanib for the treatment of medullary thyroid cancer
Nicole G Chau1, Robert I Haddad
1Medical Oncology, Head and Neck Oncology Program, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA.
Abstract:
Vandetanib (ZD6474, Caprelsa, AstraZeneca), an oral small-molecule tyrosine kinase inhibitor (TKI) that targets the rearranged during transfection receptor (RET), VEGF receptor (VEGFR2-3), and EGF receptor (EGFR), is the first systemic therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of symptomatic or progressive advanced medullary thyroid cancer (MTC). In a randomized phase III trial of patients with unresectable, locally advanced, or metastatic MTC, vandetanib improved progression-free survival compared with placebo [HR, 0.46; 95% confidence interval (CI), 0.31-0.69; P < 0.001]. However, the benefits in delaying disease progression need to be balanced against the associated and potentially serious toxicities, including diarrhea, hypertension, and QTc prolongation. Here, we review the clinical development of vandetanib leading to its integration into the current treatment paradigm and highlight the ongoing and future challenges in TKI use in MTC.
Insights
Vandetanib is the first FDA-approved systemic therapy for advanced medullary thyroid cancer (MTC), significantly improving progression-free survival. However, its benefits must be weighed against serious toxicities like diarrhea and hypertension.
Area of Science:
- Oncology
- Pharmacology
- Clinical Trials
Background:
- Medullary thyroid cancer (MTC) is a rare endocrine malignancy.
- Systemic therapies for advanced MTC have been limited.
- Vandetanib represents a novel targeted therapy approach.
Purpose of the Study:
- To review the clinical development and efficacy of vandetanib for advanced MTC.
- To evaluate the benefit-risk profile of vandetanib in MTC treatment.
- To discuss challenges in tyrosine kinase inhibitor (TKI) use for MTC.
Main Methods:
- Phase III randomized trial comparing vandetanib to placebo in advanced MTC patients.
- Analysis of progression-free survival (PFS) as the primary endpoint.
- Assessment of adverse events associated with vandetanib treatment.
Main Results:
- Vandetanib significantly improved PFS compared to placebo (HR, 0.46; P < 0.001).
- Key toxicities include diarrhea, hypertension, and QTc prolongation.
- Vandetanib is the first systemic therapy approved for symptomatic/progressive advanced MTC.
Conclusions:
- Vandetanib offers a significant survival benefit for advanced MTC.
- Careful monitoring for toxicities is crucial for patients on vandetanib.
- Ongoing research is needed to optimize TKI therapy in MTC.
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