Medical Management of Basal Cell Carcinoma with a Small-Molecule Hedgehog Inhibitor

Two recent studies show efficacy of vismodegib for treating locally advanced or metastatic BCCs.

Most basal cell carcinomas (BCCs) are removed surgically; a few are treated with radiation or topical therapies. Mutations in the PTCH1 gene, a member of the hedgehog signal transduction pathway, are involved in most BCCs. This year, the FDA approved vismodegib, a small-molecule inhibitor of the hedgehog pathway, for treating locally advanced or metastatic BCC. Researchers tested vismodegib's efficacy and safety in two multicenter trials.

Sekulic and colleagues studied 33 metastatic and 63 inoperable, locally advanced cases of BCC. The response rate was 30% in metastatic BCC (all partial responses). The response rate in locally advanced disease was 43% (perhaps an underestimate, as scarring was included in the measurement of residual tumor).…

Reviewing Author

Craig A. Elmets, MD

Disclosures

Consultant / Advisory board Astellas Pharmaceuticals

EquityVaxin

Grant / Research support NIH; NIH/NCI; Veteran’s Administration; Ferndale Laboratories; Kyowa Hakko Kirin Pharma, Inc.

Editorial boards Cancer Prevention Research; Photodermatology, Photoimmunology, & Photomedicine; UpToDate; eMedicine; Journal of Dermatological Sciences; JAMA Dermatology

Leadership positions in professional societies American Academy of Dermatology (Vice Chair, Committee on Science and Research); Photomedicine Society (Board of Directors)