miRNAs in medullary thyroid carcinoma: when will they be relevant to the clinic?

Despite a better knowledge in the molecular signaling pathways involved in the pathogenesis of medullary thyroid carcinoma (MTC), this disease, when metastatic at diagnosis, shows an unfavorable prognostic outcome [1,2]. Recent clinical trials using specific targeted therapies have shown promising results, but the low rates of partial or complete responses and the short duration of responses in these types of patients, has prompted researchers to investigate for novel biological mechanisms of the disease and to take into account to develop alternative targeted therapies [2,3]. In this scenario, miRNAs (or miRs), are important post-transcriptional regulators of several cellular biological processes and are emerging as important diagnostic tools, prognosticators and potential targets for cancer therapy [4]. Recent studies have demonstrated the presence of several dysregulated miRNAs in different types of thyroid cancers of follicular origin [5–8]. However, only few data have been generated for MTC, which is certainly an infrequent disease and is characterized by the lack of a ‘normal’ counterpart of cells to use for comparison analyses.

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