Fresh CD19 CAR T Achieves 100% MRD-Neg CR, 83% 3-yr OS in r/r Pediatric B-ALL

A point-of-care, second-generation 4-1BB CD19 CAR T product was manufactured fresh in 12 days and infused in the Phase I/II NCT04787263 trial (n = 19) enrolling children/young adults in very-high-risk first relapse or ≥2nd relapsed/refractory B-ALL. All patients attained bone-marrow CR with undetectable MRD; grade 1-2 CRS occurred in 68% (no ≥grade 3), grade 1-2 ICANS in 11%, and no dose-limiting toxicities were seen. CR durability reached 68% with 3-year EFS 68% and OS 83%, comparing favourably to the commercial CD19 CAR T tisagenlecleucel, which reports 81% CR, 63% 3-yr OS, 44% 3-yr EFS, and ≥grade 3 CRS 48%.