Vismodegib is an orally active small molecule that inhibits the hedgehog signaling pathway by binding to and inhibiting the transmembrane protein smoothened homologue (SMO). It was discovered by high-throughput screening of a library of small-molecule compounds and subsequent optimization through medicinal chemistry. Since it targets the hedgehog signaling pathway, vismodegib has anti-tumor activity in basal-cell carcinoma. The Hedgehog signaling pathway plays an important role in the development of organs and tissues during embryogenesis. Afterwards, it is silenced in all cells and tissues, except for hair, skin and stem cells. However, dysregulated or aberrant Hedgehog signaling has been associated with basal cell carcinoma pathogenesis. In January 2012, vismodegib was approved by the FDA for the treatment of adult basal cell carcinoma. In July 2013, it was approved by the EMA, and since then, it has been approved by several other countries.
用于不能手术或放疗的局部晚期皮肤基底细胞癌患者和肿瘤已转移的患者。
Skånes Universitetssjukhus; Hudkliniken, Lund, Sweden
SU/Sahlgrenska, Hudkliniken, Göteborg, Sweden
Norrlands universitetssjukhus; Onkologkliniken, Umeå, Sweden
NYU Bluestone Center For Cllinical Research, New York, New York, United States
Huntsman Cancer Institute/University of Utah, Salt Lake City, Utah, United States
University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, United States
Johns Hopkins Hospital, Baltimore, Maryland, United States
University Of Chicago Medical Center; Section Of Hematology/Oncology, Chicago, Illinois, United States
MD Anderson, Houston, Texas, United States
University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, United States
Jewish General Hospital, Montreal, Quebec, Canada
University of Arizona Cancer Center, Tucson, Arizona, United States
CHRU de Lille _ Hôpital Huriez, Lille, France
CHU de Nantes _ Hôtel Dieu, Nantes, France
Centre Henri Becquerel, Rouen, France
Loma Linda University Medical Center, Loma Linda, California, United States
Mayo Clinic, Scottsdale, Arizona, United States
University of Minnesota, Minneapolis, Minnesota, United States
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