Deirdre J. Cohen, MD, Assistant Professor, Department of Medicine, Clinical Cancer Center, NYU Medical Oncology Associates, at the Cancer Institute at NYU Langone Medical Center, discusses some of the results demonstrated in trials that examined selective inhibition of the hedgehog pathway.

Cohen states that 100% of sporadic basal-cell carcinomas demonstrate activity in the hedgehog pathway. The agent vismodegib (GDC-0449), an investigational, oral, small-molecule agent, has shown efficacy in basal cell carcinoma (BCC) by inhibiting the hedgehog pathway, which is implicated in over 90% of BCC cases. The overall response rate to this agent was approximately 50%, with measurable variations based on whether the disease was locally advanced or metastatic.

BCC is one example where hedgehog inhibition is very successful, however in epithelial malignancies it has failed to demonstrate any efficacy. Investigations are still underway to determine if this pathway plays a role in gastroesophageal cancer.