Pilot clinical trial of DNA vaccination against neuroblastoma: study design and preliminary results

Abstract

Introduction. We report preliminary results of a pilot clinical trial of therapeutic DNA vaccination of patients with neuroblastoma (NCT04049864).The aim of the study – is to test the safety and immunogenicity of DNA vaccination against neuroblastoma.Materials and methods. The results of 6 patients who completed vaccination are summarized in the article. Inclusion and exclusion criteria for patients are defined. The clinical protocol included vaccine form and doses, timed vaccination regimen, and concomitant therapy. Minimal residual disease was analyzed for all patients by quantitative polymerase chain reaction, measurement of T-cell immune response by ELISpot and antisense response by ELISA.Results. The vaccine was well tolerated by patients with minimal adverse symptoms. T-cell immune response was evaluated two weeks after each course of vaccination and was positive in 5 of 6 patients. An antisense immune response was detected in 1 patient. 5 out of 6 patients are alive and in clinical remission as of 11/01/2022. Event-free survival of vaccinated patients was 82 ± 18 % vs 29 ± 11 % of controls (p = 0.03).