A phase 2 trial found that neoadjuvant cemiplimab (Libtayo) produced encouraging results in 35 patients with locally advanced head and neck basal cell carcinoma who had not received Hedgehog inhibitor therapy. The treatment achieved a 60.1% objective response rate and a 91.4% disease control rate. Responding patients had an average tumor reduction of 70%, with 40% achieving a major or complete pathologic response.
The immunotherapy was generally well tolerated. About 35% of patients experienced treatment-related side effects, most commonly mild itching and fatigue. Only one patient stopped treatment because of a severe side effect.
Researchers also reported a 42.9% surgical benefit rate, meaning nearly half of the patients avoided removal of critical facial structures, including the nose, eyelid, ear, lip, or eye socket. Patients who completed immunotherapy followed by surgery remained disease-free, while two who skipped surgery later experienced cancer recurrence. Larger studies are planned to confirm the findings.