(HealthDay News) – A small-molecule inhibitor of the hedgehog pathway, vismodegib, is associated with tumor response in patients with metastatic or locally advanced basal cell carcinoma (BCC); and vismodegib is efficacious for preventing and treating BCCs in patients with basal cell nevus syndrome, according to two studies published in the June 7 issue of the New England Journal of Medicine.

Aleksandar Sekulic, MD, PhD, from the Mayo Clinic in Scottsdale, AZ, and colleagues enrolled 33 patients with metastatic BCC and 63 with locally advanced BCC in a multicenter, international, two-cohort, nonrandomized study. Participants received 150mg oral vismodegib daily. The researchers found that the objective response rate was 30% in the patients with metastatic BCC and 43% in those with locally advanced BCC, with complete responses in 21% of patients in the cohort with locally advanced BCC. In both cohorts, the median duration of response was 7.6 months.

Jean Y. Tang, MD, PhD, of the Children’s Hospital of Oakland Research Institute in California, and colleagues conducted a randomized, double-blind, placebo-controlled trial of vismodegib involving 41 patients with basal cell nevus syndrome. After a mean follow-up of eight months, the researchers found that the per-patient rate of new surgically eligible BCCs was significantly lower with vismodegib than placebo. The size of existing clinically significant BCCs was also significantly smaller with vismodegib. Due to adverse events, 54% of patients receiving the drug discontinued vismodegib treatment.

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“Our findings confirm the essential role of the hedgehog pathway in basal cell carcinomas and indicate that vismodegib is efficacious in preventing and treating basal cell carcinomas in patients with the basal cell nevus syndrome,” Tang and colleagues conclude.

Authors from both studies disclosed financial ties to pharmaceutical companies, including Genentech, which funded the studies and developed vismodegib.

Full Text – Sekulic (subscription or payment may be required)
Full Text – Tang (subscription or payment may be required)
Editorial (subscription or payment may be required)