TY - JOUR T1 - Basal cell carcinoma: PD-L1/PD-1 checkpoint expression and tumor regression after PD-1 blockade JF - Journal for ImmunoTherapy of Cancer JO - J Immunother Cancer DO - 10.1186/s40425-017-0228-3 VL - 5 IS - 1 SP - 23 AU - Evan J. Lipson AU - Mohammed T. Lilo AU - Aleksandra Ogurtsova AU - Jessica Esandrio AU - Haiying Xu AU - Patricia Brothers AU - Megan Schollenberger AU - William H. Sharfman AU - Janis M. Taube Y1 - 2017/12/01 UR - http://jitc.bmj.com/content/5/1/23.abstract N2 - Monoclonal antibodies that block immune regulatory proteins such as programmed death-1 (PD-1) have demonstrated remarkable efficacy in controlling the growth of multiple tumor types. Unresectable or metastatic basal cell carcinoma, however, has largely gone untested. Because PD-Ligand-1 (PD-L1) expression in other tumor types has been associated with response to anti-PD-1, we investigated the expression of PD-L1 and its association with PD-1 expression in the basal cell carcinoma tumor microenvironment. Among 40 basal cell carcinoma specimens, 9/40 (22%) demonstrated PD-L1 expression on tumor cells, and 33/40 (82%) demonstrated PD-L1 expression on tumor-infiltrating lymphocytes and associated macrophages. PD-L1 was observed in close geographic association to PD-1+ tumor infiltrating lymphocytes. Additionally, we present, here, the first report of an objective anti-tumor response to pembrolizumab (anti-PD-1) in a patient with metastatic PD-L1 (+) basal cell carcinoma, whose disease had previously progressed through hedgehog pathway-directed therapy. The patient remains in a partial response 14 months after initiation of therapy. Taken together, our findings provide a rationale for testing anti-PD-1 therapy in patients with advanced basal cell carcinoma, either as initial treatment or after acquired resistance to hedgehog pathway inhibition.Abbreviations:BCCBasal cell carcinomaHhHedgehogPD-1Programmed Death-1PD-L1Programmed Death Ligand-1 ER -