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Background: Anaplastic thyroid cancer (ATC) is a rare but highly aggressive malignancy with a median survival of 3–5 months. The BRAF oncogene is mutated to its active form in up to 24% of ATC cases. Sorafenib is a tyrosine kinase inhibitor that acts on the RAF-1 serine/threonine kinase. In preclinical mouse models, sorafenib inhibits the growth of ATC xenografts and improves survival. No study of sorafenib in ATC has been conducted. We conducted a multi-institutional phase II trial of sorafenib in patients with ATC who had failed up to two previous therapies.

Methods: The primary endpoint of the trial was the Response Evaluation Criteria In Solid Tumors (RECIST)–defined imaging response rate. Twenty patients with ATC were treated with sorafenib 400 mg twice daily.

Results: Two of the 20 patients had a partial response (10%) and an additional 5 of 20 (25%) had stable disease. The duration of response in …