Phase 1 trial of concurrent intrathecal and intravenous nivolumab for melanoma with leptomeningeal disease

Recent breakthroughs with systemic therapies have shown encouraging results for melanoma patients with extracranial diseas only, as well as parenchymal melanoma brain metastases. Howvere, there has been little progress for melanoma patients with leptomeningeal disease (LMD). A phase 1 trial at MD Anderson Cancer Center (Houston, TX) evaluated concurrent intrathecal (IT) and intravenous (IV) nivolumab for melanoma patients with LMD. There were no dose-limiting toxicities (highest dose level 50 mg IT nivolumab every two weeks). Median overall survival was 4.9 months, with 44% and 26% OS rates at 26 and 52 weeks, respectively. The authors conclude that these initial results suggest that concurrent IT and IV nivolumab is safe and feasible with potential efficacy in patients with melanoma and LMD.