Data from Targeting Superficial or Nodular Basal Cell Carcinoma with Topically Formulated Small Molecule Inhibitor of Smoothened

    March 2023
    Tracy Tang, Jean Y. Tang, Dongwei Li, Mike Reich, Christopher A. Callahan, Ling Fu, Robert L. Yauch, Frank Wang, Karen Kotkow, Kris S. Chang, Elana Shpall, Sean M. Wu, Lee L. Rubin, James C. Marsters, Ervin Epstein, Ivor Caro, Frédéric J. de Sauvage
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    TLDR The treatment was ineffective in humans.
    The study investigated the efficacy of CUR61414, a topical inhibitor of the Hedgehog signaling molecule Smoothened, in treating basal cell carcinomas (BCC). Preclinical tests on mice showed that CUR61414 significantly inhibited Hedgehog signaling, blocked hair follicle growth, and reduced BCC size. However, in a phase I clinical trial involving human subjects with superficial or nodular BCCs, the treatment showed no clinical activity. The results underscore the difficulties in translating successful preclinical findings into effective human treatments.
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