- Zak, Jaroslav;
- Pratumchai, Isaraphorn;
- Marro, Brett S;
- Marquardt, Kristi L;
- Zavareh, Reza Beheshti;
- Lairson, Luke L;
- Oldstone, Michael BA;
- Varner, Judith A;
- Hegerova, Livia;
- Cao, Qing;
- Farooq, Umar;
- Kenkre, Vaishalee P;
- Bachanova, Veronika;
- Teijaro, John R
Unleashing antitumor T cell activity by checkpoint inhibitor immunotherapy is effective in cancer patients, but clinical responses are limited. Cytokine signaling through the Janus kinase (JAK)-signal transducer and activator of transcription (STAT) pathway correlates with checkpoint immunotherapy resistance. We report a phase I clinical trial of the JAK inhibitor ruxolitinib with anti-PD-1 antibody nivolumab in Hodgkin lymphoma patients relapsed or refractory following checkpoint inhibitor immunotherapy. The combination yielded a best overall response rate of 53% (10/19). Ruxolitinib significantly reduced neutrophil-to-lymphocyte ratios and percentages of myeloid suppressor cells but increased numbers of cytokine-producing T cells. Ruxolitinib rescued the function of exhausted T cells and enhanced the efficacy of immune checkpoint blockade in preclinical solid tumor and lymphoma models. This synergy was characterized by a switch from suppressive to immunostimulatory myeloid cells, which enhanced T cell division.