Hedgehog inhibitors: a patent review (2013-present)

Introduction: The hedgehog (Hh) signaling pathway is an important signaling pathway, playing a critical role in regulation of cell growth, development, metastasis and angiogenesis. Aberrant activation of this pathway has been linked to the development of several human tumor types, which makes it an attractive target for cancer treatment. Now many efforts in both industry and academia have been made to explore small molecular Hh inhibitors as anticancer agents. Areas covered: This review aims to provide an overview of recent patents (2013 - present) in the discovery, research and development of Hh inhibitors. Expert opinion: Hh signaling pathway inhibitors attract much interest for their therapeutic potential in the treatment of a variety of human cancers. In 2012, vismodegib was approved by the FDA as a smoothened inhibitor for locally advanced or metastatic basal cell carcinoma treatment, and presently, it is ongoing clinical trials for validating its use in other tumor types. It is clear that Hh inhibitors may provide a promising clinical benefit in treating tumors involving active Hh signaling pathway with a mutation-driven mechanism. However, the efficacy of Hh inhibitors on other human tumor types is still needed to further identify.