Hedgehog antagonist GDC‐0449 is effective in the treatment of advanced basal cell carcinoma

Objectives/Hypothesis: To demonstrate the efficacy of the hedgehog pathway inhibitor GDC‐0449 in the treatment of advanced basal cell carcinoma. Design Study: Case series. Methods: Three patients treated in a referral center for locally advanced basal cell carcinoma, one with metastases, were referred for treatment in a GDC‐0449 phase I clinical trial. The treatment was once per day continuous therapy with oral GDC‐0449. Results: Two patients showed complete clinical and radiologic resolution of disease, whereas one patient had significant reduction in tumor burden with radiologic evidence of slowly progressive local disease. Side effects were taste changes, mild to moderate hair loss, and muscle cramps in one patient. Conclusions: GDC‐0449 showed significant inhibitory activity in the treatment of advanced basal cell carcinoma.