Efficacy and safety of autologous <scp>CIK</scp> cell therapy plus Toripalimab with or without chemotherapy in advanced <scp>NSCLC</scp>: A phase <scp>II</scp> study

Advanced non-small cell lung cancer (NSCLC) with positive PD-L1 expression requires more effective therapeutic options. This study aims to evaluate the efficacy and safety of autologous cytokine-induced killer (CIK) cell therapy combined with the anti-PD-1 antibody toripalimab, with or without chemotherapy, as a first-line treatment for advanced NSCLC. This phase II trial enrolled 40 patients with PD-L1-positive, driver mutation-negative advanced NSCLC between July 2020 and December 2022. Patients were randomly assigned to Arm A (toripalimab + CIK cells + chemotherapy) or Arm B (toripalimab + CIK cells). Progression-free survival (PFS), overall survival (OS), overall response rate (ORR), and safety profiles were evaluated. Subgroup analyses were conducted based on the number of CIK cell cycles received. Arm A showed a significantly longer median PFS compared to Arm B (20.0 vs. 6.0 months, p = 0.0038), while median OS was not reached in Arm A versus 17.0 months in Arm B (p = 0.0479). ORR was 47.4% in Arm A and 60.0% in Arm B. Patients receiving four or more cycles of CIK cells had significantly improved PFS and OS. No new safety concerns were identified. The combination of CIK cells and toripalimab, with or without chemotherapy, demonstrates promising efficacy and safety in patients with advanced PD-L1-positive NSCLC. The addition of chemotherapy may further enhance therapeutic outcomes, making it a potentially superior strategy compared to CIK cells combined with the anti-PD-1 antibody alone.