Recent advances in precision medicine for the treatment of medullary thyroid cancer

Abstract

ABSTRACT Introduction The discovery of a pivotal role of tyrosine kinases in the pathogenesis of medullary thyroid carcinoma (MTC) opened up new options in the systemic treatment of advanced disease. During the last decade, two multikinase inhibitors (MKIs) – vandetanib and cabozantinib and, more recently, two potent, selective RET inhibitors selpercatinib and pralsetinib gained regulatory approval. Areas covered The data on efficacy and safety of vandetanib, cabozantinib, selpercatinib, and pralsetinib in MTC published during the recent 10 years. Expert opinion The perspectives of systemic MTC treatment have substantially changed since 2010. However, to date, the question of which drug should be chosen as the first line remains open. There are no recommendations on what to use as the second or other treatment lines. The impact of tumor burden, progression slope, and the presence of MTC symptoms on treatment-decision making is not unequivocally defined. Another important problem is the treatment duration. EBM (evidence-based medicine) study, resolving these issues, is our task for the nearest future. The treatment tolerability and its impact on the quality of life, particularly regarding nonselective MKIs, is also an essential problem.