Advanced basal cell carcinoma of the skin: Targeting the hedgehog pathway

PURPOSE OF REVIEW: This article provides an update on basal cell carcinoma (BCC), with a focus on the advanced BCC (aBCC), and the recent progress with targeted hedgehog signaling pathway inhibition for treatment of aBCC. RECENT FINDINGS: The hedgehog signaling pathway is aberrantly activated in most BCC tumors providing an attractive therapeutic target in this cancer. Recently developed targeted hedgehog pathway inhibitors have demonstrated remarkable efficacy in the treatment of aBCC and the first oral hedgehog pathway inhibitor vismodegib was granted the US Food and Drug Administration (FDA) approval. Toxicities of the current hedgehog pathway inhibitors are mostly mild to moderate, but with prolonged treatment can pose a therapeutic challenge. SUMMARY: Hedgehog pathway inhibition is a novel and powerful approach for treatment of aBCC. Current research efforts aim to enhance the activity and minimize toxicity of this promising new therapy.