Meiss Frank, Andrlová Hana, Zeiser Robert
Department of Dermatology and Venereology, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Hauptstr. 7, 79104, Freiburg, Germany.
Department of Hematology, Oncology and Stem Cell Transplantation, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Hugstetter Str. 55, 79106, Freiburg, Germany.
Recent Results Cancer Res. 2018;211:125-139. doi: 10.1007/978-3-319-91442-8_9.
Vismodegib (GDC-0449, Erivedge) is a small molecule antagonist of the hedgehog (Hh) pathway that binds to smoothened (SMO) and leads to inhibition of an aberrant activation of the Hh pathway. Dysregulated Hh signaling results in uncontrolled proliferation in basal cell carcinoma (BCC) and has also been found present in medulloblastoma, and many other cancers such as those of gastrointestinal tract, brain, lung, breast, and prostate. In January 2012, vismodegib became the first agent to target the Hh pathway to receive approval by the United States Food and Drug Administration (FDA) and in July 2013 approval by the European Medicines Agency (EMA) followed for the treatment of adult patients with symptomatic metastatic BCC, or locally advanced BCC inappropriate for surgery or radiotherapy. The role of vismodegib in other malignancies than BCC has still to be investigated.
维莫德吉（GDC-0449，Erivedge）是一种刺猬信号通路（Hh）的小分子拮抗剂，它与 smoothened（SMO）结合，从而抑制Hh通路的异常激活。Hh信号失调会导致基底细胞癌（BCC）中细胞不受控制地增殖，并且在髓母细胞瘤以及许多其他癌症（如胃肠道、脑、肺、乳腺和前列腺癌）中也发现存在这种情况。2012年1月，维莫德吉成为首个靶向Hh通路并获得美国食品药品监督管理局（FDA）批准的药物，2013年7月，欧洲药品管理局（EMA）也批准其用于治疗有症状的转移性BCC成年患者，或不适用于手术或放疗的局部晚期BCC患者。维莫德吉在BCC以外的其他恶性肿瘤中的作用仍有待研究。
