Vandetanib for Locally Advanced or Metastatic Medullary Thyroid Cancer (MTC)

Medullary thyroid cancer (MTC) can usually be treated with surgery. However, treatment options for patients with unresectable, recurrent or metastatic MTC are limited. Mutations in the RET gene are common in sporadic MTC with the most common mutation (M918T), occurring in 92% of patients tested. Furthermore, this mutation is associated with a poor prognosis. Vandetanib is a small molecule inhibitor of three oncogenes common to MTC (RET, VEGFR2 and EGFR) and is a promising new treatment option for MTC. In an ongoing clinical trial 45% of the patients responding to vandetanib and the estimated progression free survival rate (not yet achieved), was 30.5 mths compared with 19.3 mths for the control group. Furthermore, vandetanib produced higher response rates when patients where stratified based on the type and presence or absence of RET mutations. The response rate for vandetanib in patients harbouring the M918T mutation was 54.5% compared to 32% in patients who did not have RET mutations, or for whom mutation status was unknown. I like vandetanib because it targets multiple oncogenic signaling pathways, is specific for a poor prognosis mutation commonly to sporadic MTC, has limited side effects compared with standard chemo/radiation therapy and provides new treatment options for a rare form of cancer. The following article is an excellent overview on the use of vandetanib for treating MTC.
Progress in Molecular Targeted Therapy for Thyroid Cancer: Vandetanib in Medullary Thyroid Cancer.

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