Department of Biochemistry and Molecular Biology, University of Nebraska Medical Center, Omaha, NE, USA.
Cancer Manag Res. 2013 Jul 31;5:197-203. doi: 10.2147/CMAR.S45976. Print 2013.
Basal cell carcinoma (BCC) is the most common human malignancy. Recent advances in our understanding of the critical biologic pathways implicated in the development and progression of BCC have led to the development of the first molecular targeted therapy for this disease. The hedgehog pathway is mutated in virtually all patients with BCC and recent trials with vismodegib, an inhibitor of this pathway, have shown significant responses. This review will discuss the importance of the hedgehog pathway in the pathogenesis of BCC and describe in detail the pharmacology of vismodegib in relation to its activity in advanced BCC.
基底细胞癌（BCC）是最常见的人类恶性肿瘤。近年来，我们对涉及 BCC 发生和发展的关键生物学途径的理解取得了进展，这导致了针对这种疾病的第一种分子靶向治疗方法的发展。 hedgehog 通路在几乎所有的 BCC 患者中都发生了突变，最近使用 hedgehog 通路抑制剂 vismodegib 的试验显示出了显著的疗效。这篇综述将讨论 hedgehog 通路在 BCC 发病机制中的重要性，并详细描述 vismodegib 的药理学及其在晚期 BCC 中的活性。
