The outcomes for patients with previously treated advanced stage non–small lung cancer (NSCLC) are very poor, with a modest benefit from chemotherapy over best supportive care. Immunotherapy offers a novel approach for the treatment of these patients, with two anti-programmed death 1 (PD-1) checkpoint inhibitors, nivolumab and pembrolizumab, recently approved by the FDA based on large randomized clinical trials showing increased overall survival compared with standard second-line docetaxel. Although only a subset of patients benefit from these drugs, the treatment is usually well tolerated and the responses are often durable, with an unprecedented number of survivors 3 years after starting the therapy. The next steps should be the identification of reliable predictors for benefit from immunotherapy with checkpoint inhibitors and the pursuit of well-designed combination therapies. In this article, we review the rationale for the use of checkpoint inhibitors in NSCLC, the data from phase I and randomized clinical trials, and future directions. Clin Cancer Res; 22(15); 3713–7. ©2016 AACR.
- Received March 9, 2016.
- Revision received April 26, 2016.
- Accepted April 27, 2016.
- ©2016 American Association for Cancer Research.