Low-Dose Nivolumab Injections Shrink High-Risk Oral Precancers in Early Trial

Overview

• MD Anderson researchers presented a phase I, single-arm study at AACR 2026 testing intralesional nivolumab in 29 patients with high-risk precancerous oral lesions.
• Patients received 10 or 20 mg injected into one lesion every three weeks for four cycles, producing an 85% clinical response with an average 60% drop in lesion area.
• Pathology improved in many cases, with 41% showing downgrading of dysplasia and six complete pathologic responses with no dysplasia detected on follow-up.
• At 12 months, 82% of treated sites remained cancer-free, and most patients avoided surgery that can damage speech, swallowing, and quality of life.
• Blood drug levels were about tenfold lower than typical intravenous dosing with no dose-limiting toxicities, immune activation appeared only in treated lesions, and a placebo-controlled phase II is planned to validate these findings.