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Spotlight on Clinical Response

Medullary thyroid cancer: targeting the RET kinase pathway with sorafenib/tipifarnib

¹ Department of Investigational Cancer Therapeutics (Phase I Program), Division of Cancer Medicine, ² Department of Internal Medicine, and ³ Pathology, M. D. Anderson Cancer Center, Houston, Texas; and ⁴ Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland

Requests for reprints: David Hong, Department of Investigational Therapeutics (Phase I Program), Unit 455, Division of Cancer Medicine, M. D. Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030. Phone: 713-563-5844; Fax: 713-792-0305. E-mail: dshong{at}mdanderson.org

Abstract

Medullary thyroid carcinoma (MTC) is an uncommon malignancy of hereditary and sporadic presentation. Mutations in the RET proto-oncogene are involved in the pathogenesis of familial MTC and >50% of the sporadic cases. Currently, there is no effective treatment for recurrent or metastatic MTC. We report here a rapid response to a sorafenib (RET and RAF kinase and vascular endothelial growth factor receptor inhibitor)–based regimen in a patient with sporadic MTC who had advanced, progressive disease and a novel RET kinase aberration at exon 11 shown in tumor tissue. [Mol Cancer Ther 2008;7(5):1001–6]

Grant support: NIH grant 5 UO1 CA062461 (R. Kurzrock) and Translational Research Initiative grant 25XS068 (D. Hong).

The costs of publication of this article were defrayed in part by the payment of page charges. This article must therefore be hereby marked advertisement in accordance with 18 U.S.C. Section 1734 solely to indicate this fact.

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Received 12/21/07; accepted 2/26/08.