Data from a phase 1/2 study presented at the EBMT 52nd Annual Meeting shows that rapcabtagene autoleucel (YTB323), a next-generation CAR T-cell therapy, is highly effective for adults with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).
After a median follow-up of 27.7 months, 86% of patients responded to treatment within three months. All responders achieved deep remission with no detectable cancer cells. Higher dose levels showed more durable responses, with over half of patients maintaining remission at one year.
The treatment also showed expected but manageable side effects. Cytokine release syndrome occurred in most patients, with some severe cases, while neurotoxicity was less common. Nearly all patients experienced low blood cell counts, which is typical for this type of therapy.
The study included 35 heavily pretreated adults who had received multiple prior therapies. Patients underwent immune system preparation, followed by manufacturing of personalized CAR T-cells and a single infusion at different dose levels.
Overall, the results support further development of rapcabtagene autoleucel as a powerful chemotherapy-free option for patients who have limited treatment choices.