Spotlight on Clinical Response

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

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

^* To whom correspondence should be addressed. 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):OF1–6]

Key Words: cancer, carcinoma malignancy, Ras, Raf, RET, tipifarnib, sorafenib, squamous cell cancer, Phase I, kinase