First-in-human phase I clinical trial of NY-ESO-1 protein cancer vaccine with a novel adjuvant MIS416, NOD2 and TLR9 stimulant, for patients with NY-ESO-1 expressing solid tumors.

e15176Background: Complexes of Cholesteryl pullulan and NY-ESO-1 antigen (CHP-NY-ESO-1) present multiple epitope peptides to both the MHC class I and class II pathways. MIS416 is a non-toxic microparticle adjuvant that activates immune responses via NOD-2 and TLR9 pathways. Vaccination studies in mice have demonstrated that CHP-NY-ESO-1 with MIS416 enhanced anti-tumor efficacy. We conducted a First-in-Human clinical trial using CHP-NY-ESO-1 with adjuvant MIS416 for patients (pts) with NY-ESO-1-expressing refractory solid tumor (Clinical trial registration number, UMIN000005246, UMIN000008007). Methods: NY-ESO-1 expression was assessed by immunohistochemistry and/or RT-PCR. Pts received CHP-NY-ESO-1/MIS416 (C/M) 100/200, 200/200, 200/400 or 200/600 (μg/μg) (every 2 weeks, 6 doses total) followed by one vaccination every 4 weeks, until disease progression or unacceptable toxicity was observed. Response was assessed every 12 weeks by CT. The primary endpoints were safety and tolerability, and the secondary e...