Basal Cell Carcinoma, Hedgehog Signaling, and Targeted Therapeutics: The Long and Winding Road

The document reviewed the role of Hedgehog (Hh) signaling in the pathogenesis of basal cell carcinoma (BCC) and the development of targeted therapeutics, such as vismodegib, to inhibit this pathway. It discussed the genetic mutations, particularly in the PTCH1 gene, that predispose individuals to BCC and the effectiveness of Hh pathway inhibitors in treating locally advanced and metastatic BCC, with response rates of 43% and 30% respectively. The review also mentioned the potential for drug resistance and side effects like muscle cramps and alopecia. While Hh pathway inhibitors represent a significant advancement in treating advanced BCC, their effectiveness in less advanced cases or as a preventative treatment is still uncertain, and further research is needed to understand and mitigate the side effects of these drugs.