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

    May 2011 in “ Clinical cancer research
    Tracy Tang, Jean Y. Tang, Dongwei Li, Mike Reich Reich, Christopher A. Callahan, Ling Fu, Robert L. Yauch, Frank Wang, Karen Kotkow, Kris S. Chang, Elana Shpall, Angela Ruohao Wu, Lee L. Rubin, James C. Marsters, Ervin Epstein, Ivor Caro, Frédéric J. de Sauvage
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    TLDR The topical inhibitor CUR61414 was not effective in treating basal cell carcinoma in human trials.
    In a study from 2011, researchers investigated the effectiveness of CUR61414, a topical inhibitor of the Smoothened molecule, which is part of the Hedgehog (Hh) signaling pathway implicated in the development of basal cell carcinomas (BCC). Preclinical studies in mice showed that CUR61414 could inhibit Hh signaling in the skin, prevent hair follicle growth, and reduce BCC size. However, in a phase I clinical trial involving human patients with superficial or nodular BCCs, the topical application of CUR61414 at various concentrations (0.09%, 0.35%, 1.1%, and 3.1%) for up to 28 days did not demonstrate clinical efficacy. This discrepancy between animal models and human trials underscores the challenges in translating preclinical findings into effective human treatments.
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