Understanding BCC pathogenesis: Treatment advancements and challenges

Basal cell carcinoma (BCC) is generally a slow growing tumor, but an important subset of BCC patients develop locally advanced or, more rarely, metastatic disease. Overactivity of the hedgehog (HH) signaling pathway has emerged as a major driver of BCCs in general, leading to recent Food and Drug Administration (FDA) approval of a HH inhibitor (vismodegib) for the treatment of patients with metastatic or locally advanced BCC that has recurred following surgery or who are not candidates for radiotherapy. This represents a major advance for an important subset of patients with previously limited and ineffective treatment options. Additional HH inhibitors and other targeted agents are currently in development. How best to utilize vismodegib and other emerging HH inhibitors for BCC is a topic of discussion and further study.