A phase I/II trial: WU-CART-007 for relapsed/refractory T-ALL or LBL

Results from a phase I/II trial (NCT04984356), evaluating the safety and efficacy of WU-CART-007, a CD7-targeting chimeric antigen receptor (CAR) T-cell therapy in patients with relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) or lymphoblastic leukemia (LBL), were published by Ghobadi et al. in Blood. Of 28 patients enrolled, 13 received the recommended phase II dose (RP2D) of 900 × 10⁶ cells.

Key data: Cytokine release syndrome (CRS) was the most common treatment-related adverse event (TRAE), occurring in 88.5% of all patients, of whom 19.2% experienced Grade 3–4 CRS. Grade 1 immune effector cell-associated neurotoxicity syndrome (ICANS) was observed in 7.7% of patients, while Grade 2 acute graft-versus-host disease (aGvHD) occurred in 3.8%. Among the evaluable patients for response at the recommended phase II dose (RP2D) who received enhanced lymphodepleting chemotherapy (n = 11), the overall response rate (ORR) was 90.9% with a composite complete remission (CRc) rate of 72.7%. 

Key learning: At the RP2D, WU-CART-007 achieved a high response rate with a manageable safety profile, supporting its potential as a novel treatment option for R/R T-cell malignancies and addressing an unmet need in this population.