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Updated: Jan 23, 2026

A Simple Bioassay for the Evaluation of Vascular Endothelial Growth Factors
Published on: March 15, 2016
Phase 2 study of vascular endothelial growth factor trap for the treatment of metastatic thyroid cancer
Eric J Sherman1,2, Lara A Dunn1,2, Heiko Schöder3
1Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York.
Background:
Several multitargeted tyrosine kinase inhibitors (TKIs) have demonstrated activity in patients with thyroid cancer that is refractory to radioactive iodine (RAI). The antitumor effect is attributed at least in part to the ability of these TKIs to inhibit angiogenesis in these vascular tumors. Vascular endothelial growth factor (VEGF) Trap (VT) is a recombinantly produced fusion protein consisting solely of human sequences for VEGF receptors 1 and 2 extracellular domains and human immunoglobulin 1. Evaluating VT in patients with thyroid cancer is reasonable considering the activity observed with TKIs targeting VEGF.
Methods:
The current study was a single-institution, phase 2, Simon 2-stage design (21 to >41 patients) study based on the objective response rate and/or 6-month progression-free survival as the primary endpoints. Eligible patients were required to have progressive, RAI-refractory and/or [18 F]fludeoxyglucose-avid, recurrent and/or metastatic, nonmedullary, nonanaplastic thyroid cancer; disease that was measurable using Response Evaluation Criteria In Solid Tumors (RECIST) criteria; and adequate organ and bone marrow function. VT at a dose of 4 mg/kg intravenously was administered every 14 days.
Results:
A total of 40 patients were included in the analysis. Of these patients, 24 had papillary thyroid cancer, 2 had follicular thyroid cancer, and 11 had Hurthle cell thyroid cancer. The final 3 tumors were classified as poorly differentiated. There were no complete and/or partial responses noted; 34 patients achieved stable disease and 6 patients experienced disease progression as their best response. Of the 34 patients with stable disease, 16 remained on the study for >6 months and 6 patients remained on the study for >12 months. The median duration on treatment was 4.1 months (range, 0.6-30.8 months).
Conclusions:
Unlike TKIs, which have shown responses in this setting, to the authors' knowledge there have been no responses observed with the use of single-agent VT to date. It does not appear to be a promising drug for the treatment of patients with thyroid cancer.
Insights
Vascular Endothelial Growth Factor (VEGF) Trap did not show significant responses in patients with advanced thyroid cancer. This study suggests VEGF Trap is not a promising treatment for this condition.
Area of Science:
- Oncology
- Pharmacology
- Clinical Trials
Background:
- Multitargeted tyrosine kinase inhibitors (TKIs) show activity in radioactive iodine-refractory thyroid cancer, partly by inhibiting angiogenesis.
- Vascular Endothelial Growth Factor (VEGF) Trap (VT) targets VEGF, a key factor in tumor vascularization.
Purpose of the Study:
- To evaluate the efficacy of single-agent VEGF Trap in patients with progressive, radioactive iodine-refractory thyroid cancer.
- To assess objective response rate and 6-month progression-free survival as primary endpoints.
Main Methods:
- Phase 2, single-institution, Simon 2-stage design study.
- 40 patients with progressive, RAI-refractory, recurrent/metastatic nonmedullary thyroid cancer received VEGF Trap (4 mg/kg IV every 14 days).
- Disease measured by RECIST criteria.
Main Results:
- No complete or partial responses were observed in 40 patients.
- 34 patients achieved stable disease; 6 experienced disease progression.
- Median treatment duration was 4.1 months; 16 patients had stable disease for over 6 months.
Conclusions:
- Single-agent VEGF Trap demonstrated no objective responses in this patient cohort.
- VEGF Trap does not appear to be a promising therapeutic option for advanced thyroid cancer.
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