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Published on: June 2, 2023
Axitinib is an active treatment for all histologic subtypes of advanced thyroid cancer: results from a phase II study
Ezra E W Cohen1, Lee S Rosen, Everett E Vokes
1Section of Hematology/Oncology, Department of Medicine, University of Chicago Division of Biological Sciences, 5801 S Ellis, Chicago, IL 60637, USA. ecohen@medicine.bsd.uchicago.edu
Purpose:
Patients with advanced, incurable thyroid cancer not amenable to surgery or radioactive iodine ((131)I) therapy have few satisfactory therapeutic options. This multi-institutional study assessed the activity and safety of axitinib, an oral, potent, and selective inhibitor of vascular endothelial growth factor receptors (VEGFR) 1, 2, and 3 in patients with advanced thyroid cancer.
Patients And Methods:
Patients with thyroid cancer of any histology that was resistant or not appropriate for (131)I were enrolled onto a single-arm phase II trial to receive axitinib orally (starting dose, 5 mg twice daily). Objective response rate (ORR) by Response Evaluation Criteria in Solid Tumors was the primary end point. Secondary end points included duration of response, progression-free survival (PFS), overall survival, safety, and modulation of soluble (s) VEGFR.
Results:
Sixty patients were enrolled. Partial responses were observed in 18 patients, yielding an ORR of 30% (95% CI, 18.9 to 43.2). Stable disease lasting > or = 16 weeks was reported in another 23 patients (38%).
Objective:
responses were noted in all histologic subtypes. Median PFS was 18.1 months (95% CI, 12.1 to not estimable). Axitinib was generally well tolerated, with the most common grade > or = 3 treatment-related adverse event being hypertension (n = 7; 12%). Eight patients (13%) discontinued treatment because of adverse events. Axitinib selectively decreased sVEGFR-2 and sVEGFR-3 plasma concentrations versus sKIT, demonstrating its targeting of VEGFR.
Conclusion:
Axitinib is a selective inhibitor of VEGFR with compelling antitumor activity in all histologic subtypes of advanced thyroid cancer.
Insights
Axitinib shows significant antitumor activity in advanced thyroid cancer patients resistant to radioactive iodine therapy. This oral vascular endothelial growth factor receptor inhibitor demonstrated a 30% objective response rate and was generally well tolerated.
Area of Science:
- Oncology
- Pharmacology
Background:
- Advanced, incurable thyroid cancer lacks effective treatments, especially for patients unresponsive to surgery or radioactive iodine ((131)I).
- Vascular Endothelial Growth Factor Receptors (VEGFR) are key targets in angiogenesis and cancer progression.
Purpose of the Study:
- To assess the efficacy and safety of axitinib, a VEGFR inhibitor, in patients with advanced thyroid cancer.
- To evaluate axitinib's activity in thyroid cancer histologies resistant or unsuitable for (131)I therapy.
Main Methods:
- A single-arm, multi-institutional phase II trial enrolled 60 patients with advanced thyroid cancer.
- Patients received oral axitinib (5 mg twice daily).
- Primary endpoint was objective response rate (ORR); secondary endpoints included progression-free survival (PFS) and safety.
Main Results:
- Axitinib achieved an ORR of 30% (18/60 patients), with responses observed across all histologic subtypes.
- Median PFS was 18.1 months.
- Hypertension was the most common grade ≥3 adverse event (12%); 13% discontinued treatment due to adverse events.
Conclusions:
- Axitinib demonstrates compelling antitumor activity in advanced thyroid cancer, regardless of histologic subtype.
- Axitinib is a selective VEGFR inhibitor with a manageable safety profile for this patient population.
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