CAR T-Cell Therapy for ALL
Axicabtagene ciloleucel (KTE-C19) is also being investigated in the ZUMA-3 and ZUMA-4 trials for treatment of relapsed/refractory acute lymphoblastic leukemia (r/r ALL) in adult and pediatric patients.
In the Phase 1 trials, 13 patients with r/r ALL were treated with KTE-C19; eleven patients were evaluable for response. At the time of the preliminary analysis, 82% of patients (9 out of 11) achieved complete remission or complete remission with incomplete or partial hematological recovery. All responders (100%) tested negative for minimal residual disease (MRD), which is correlated with risk of disease relapse in ALL.
Adverse events grade 3 or higher included cytokine release syndrome (5 of 13 patients, 38%) and neurological events (5 of 13 patients, 38%). There were two deaths: one patient in ZUMA-3 died from KTE-C19 related cytokine release syndrome (CRS) and one patient in ZUMA-4 died from an unrelated fungal infection. Larger Phase 2 studies in r/r ALL will be ongoing in 2017.
