Phase 1 study of ipilimumab, nivolumab, or the combination in patients with newly diagnosed glioblastoma

The phase 1 NRG-BN002 trial,at nine centers in the USA, evaluated ipilimumab (anti-CTLA-4), nivolumab (anti-PD-1), and ipilimumab plus nivolumab for newly diagnosed glioblastoma (GBM) after resection and completion of standard radiochemotherapy. Thirty-two patients were enrolled; the median age was 55 years; 67.7% were male; 83.9% were white. Treatment was well tolerated with a 16% grade 4 events. The combination did not have unexpectedly increased toxicity, with no grade 5 events. There were no dose limiting toxicities with the combination. Median follow-up was 19.6 months. For patients receiving the combination treatment, median progression-free survival and overall survival were 16.1 months and 20.7 months, respectively. The authors conclude that ipilimumab and nivolumab are safe and tolerable when given in combination with adjuvant temozolomide for newly diagnosed GBM. These results support a subsequent efficacy phase 2-3 trial to test the combination for patients with newly diagnosed GBM.