A first-in-human Phase 1 study presented at the 2026 EHA Congress has reported encouraging results for CT0596, an off-the-shelf CAR T-cell therapy designed for patients with heavily pretreated relapsed or refractory multiple myeloma and primary plasma cell leukemia.
Unlike traditional CAR T-cell treatments that are made from a patient’s own cells, CT0596 uses donor cells engineered with three gene deletions to reduce the risk of graft-versus-host disease (GVHD) and immune rejection. This approach could make CAR T-cell therapy more accessible and less costly.
Among eight patients treated at the recommended dose, all responded to therapy. Seventy-five percent achieved a stringent complete response, while 87.5% achieved a very good partial response or better. All patients became minimal residual disease (MRD) negative within four weeks after treatment. After a median follow-up of nearly seven months, six patients remained in remission.
The treatment’s side effects were manageable. Most patients experienced mild cytokine release syndrome, but no cases of neurotoxicity or GVHD were reported. Temporary severe reductions in blood cell counts occurred in all patients, consistent with expectations for CAR T-cell therapy.