Randomized Phase II Trial of Dendritic Cell/AML Fusion Cell Vaccination Compared to Standard of Care Therapy in AML CR1

Abstract

Introduction: We have developed a personalized cancer vaccine in which patient derived AML cells are fused with autologous dendritic cells (DCs), presenting a broad array of antigens including shared and neoantigens. Here we report the results of the multicenter randomized phase II clinical trial (NCT03059485) of DC/AML fusion cell vaccination in AML patients who achieve a remission following chemotherapy or HMA/Venetoclax. Methods: Eligible patients were >55 years and were not candidates for allogeneic transplantation. The primary endpoint was to assess 2-year progression free survival. Leukemia cells were collected and cryopreserved at time of disease presentation. Patients underwent standard of care remission induction therapy, with either 7+3 or HMA/Ven, at the discretion of the treating physician. Patients in CR (CR/CRi) were randomized 2:1 to the vaccine (Arm A) or standard of care arm (Arm C). Arm B, vaccine with PD-1 blockade, was removed prior to any patient being treated. Patients in Arm C could receive maintenance therapy, while those in Arm A did not. Patients in Arm A underwent leukapheresis and DCs were generated by cytokine mediated differentiation. DC/AML fusions were created by co-culture of DCs and leukemia cells in the presence of polyethylene glycol. The trial was designed with a target accrual of 75 patients but closed to prematurely due to slow enrollment during COVID