Table 3.
Chimeric antigen receptor T-cell studies and their results
| Patients | Phase | N | Response | Survival | Adverse effects |
|---|---|---|---|---|---|
| Pediatric and young adults, relapsed/refractory B-ALL58 (19-BBz) |
I/II | 30 | CR*: 90% (MRD negative**: 88%) |
6-month overall survival: 78% | CRS: 100% (mild/moderate), 27% (severe) Neurologic: 43% |
| Pediatric and young adults, relapsed/refractory B-ALL59 (19-BBz) |
II | 75 | CR*: 81% (MRD negative: 100%) |
6-month overall survival: 90% | CRS: 77% (any), 46% (grade ≥3) Neurologic: 40% (any), 13% (grade 3) |
| Pediatric and young adults, relapsed/refractory B-ALL/NHL60 (19–28z) |
I | 21 | CR*: 67% (MRD negative**: 86%) |
10-month overall survival: 51.6% | CRS: 76% (any), 29% (grade ≥3) |
| Adults, relapsed/refractory B-ALL61 (19–28z) |
I | 53 | CR*: 83% (MRD negative**: 73%) |
Median survival: 12.9 months | CRS: 85% (any), 26% (grade ≥3) Neurologic: 43% (grade ≥2), 42% (grade ≥3) |
| Pediatric and young adults, relapsed/refractory B-ALL81 (19-BBz) |
I | 43 | MRD negative CR*: 93% | 12-month overall survival: 69.5% | CRS: 93% (any), 23% (severe) Neurologic: 49% (any), 21% (severe) |
| Pediatric and young adults, relapsed/refractory B-ALL101 (22-BBz) |
I | 21 | CR*: 57% (MRD negative**: 75%) CR*: 73% (≥1 × 106 CAR T) |
Median remission duration: 6 months | CRS: 76% (any), 0% (grade ≥3) No severe neurotoxicity |
| Pediatric and young adults, relapsed/refractory B-ALL80 (19 [low affinity]-BBz) |
I | 14 | MRD-negative CR*: 86% | 12-month overall survival: 63% | CRS: 93% (any), 0% (grade ≥3) Neurologic: 43% (any), 0% (grade ≥3) |
| Pediatric and adults, relapsed/refractory B-ALL/NHL103 (19 –28/BBz and 22–28/BBz cocktail) |
II | 89 | MRD-negative CR*#: 96% | 12-month overall survival#: 62.8% | CRS: 95.5% (any), 21.3% (grade ≥3) Neurologic: 13.5% (any), 1.1% (grade ≥3) |
Abbreviations: ALL, acute lymphoblastic leukemia; CR, complete remission; MRD, minimal residual disease; CRS, cytokine release syndrome; NHL, non-Hodgkin lymphoma, BBz, intracellular signaling domains of 4–1BB with CD3z; 28z, intracellular signaling domains of CD28 with CD3z
*
CR includes incomplete counts recovery
**
Percentage of MRD-negative patients among those with CR
#
ALL only (51 patients)