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Updated: May 8, 2026

Spontaneous Murine Model of Anaplastic Thyroid Cancer
Published on: February 3, 2023
Cabozantinib in progressive medullary thyroid cancer
Rossella Elisei1, Martin J Schlumberger, Stefan P Müller
1Rossella Elisei, University of Pisa, Pisa; Lisa Licitra, Fondazione Istituto Di Ricovero e Cura a Carattere Scientifico-Istituto Nazionale dei Tumori, Milan, Italy; Martin J. Schlumberger, Institut Gustave Roussy, University Paris-Sud, Villejuif, France; Stefan P. Müller, Universitatsklinikum Essen, Essen; Michael C. Kreissl, Universitätsklinikum Würzburg, Würzburg, Germany; Patrick Schöffski, University Hospitals Leuven, Leuven, Belgium; Marcia S. Brose, University of Pennsylvania Abramson Cancer Center, Philadelphia, PA; Manisha H. Shah, Ohio State University Medical Center, Columbus, OH; Barbara Jarzab, Centrum Onkologii-Instytut im. Marii Skłodowskiej-Curie Oddział w Gliwicach, Gliwice, Poland; Viktor Medvedev, Medical Radiological Research Centre of the Russian Academy of Medical Sciences, Obninsk, Russia; Bruno Niederle, Medizinische Universität Wien, Wien, Austria; Ezra E.W. Cohen, University of Chicago, Chicago, IL; Lori J. Wirth, Massachusetts General Hospital, Boston, MA; Haythem Ali, Henry Ford Health System, Detroit, MI; Colin Hessel and Yifah Yaron, Exelixis, South San Francisco, CA; Douglas Ball and Barry Nelkin, Johns Hopkins University School of Medicine, Baltimore, MD; and Steven I. Sherman, University of Texas MD Anderson Cancer Center, Houston, TX.
Cabozantinib significantly improved progression-free survival in patients with metastatic medullary thyroid cancer (MTC). This tyrosine kinase inhibitor offers a new treatment option for MTC, despite manageable side effects.
Area of Science:
- Oncology
- Pharmacology
Background:
- Medullary thyroid cancer (MTC) is a rare endocrine malignancy.
- Metastatic MTC often has a poor prognosis, necessitating novel therapeutic strategies.
Purpose of the Study:
- To evaluate the efficacy and safety of cabozantinib in patients with progressive metastatic MTC.
- To compare progression-free survival (PFS) between cabozantinib and placebo.
Main Methods:
- A double-blind, phase III trial involving 330 patients with documented radiographic progression of metastatic MTC.
- Random assignment (2:1) to cabozantinib (140 mg/day) or placebo.
- Primary endpoint: progression-free survival (PFS); secondary endpoints: tumor response rate, overall survival, and safety.
Main Results:
- Median PFS was significantly longer with cabozantinib (11.2 months) versus placebo (4.0 months) (hazard ratio, 0.28; P < .001).
- Response rate was 28% for cabozantinib compared to 0% for placebo.
- Cabozantinib demonstrated benefits across subgroups, including those with and without RET mutations.
Conclusions:
- Cabozantinib (140 mg/day) significantly improved PFS in patients with progressive metastatic MTC.
- Cabozantinib represents a new, effective treatment option for this rare cancer.
- The observed toxicity was significant but manageable.
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