Data show that rapcabtagene autoleucel (YTB323), a next-generation CAR T-cell therapy, is highly effective in adults with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).
In the Phase 1/2 study, the overall response rate reached 86% within three months. All responding patients achieved minimal residual disease (MRD) negativity, meaning no detectable cancer remained. With a median follow-up of nearly 28 months, higher dose levels were linked to longer-lasting responses.
Side effects were common but manageable. Cytokine release syndrome occurred in 86% of patients, while 14% experienced neurological toxicity. Most patients had severe adverse events, mainly low blood cell counts. Although 63% of patients died during the study, most deaths were due to disease progression rather than treatment.
The trial enrolled 35 heavily pretreated patients with a median age of 41. Lead investigator Alessandro Rambaldi said the therapy shows strong anti-cancer activity and supports further development.