Table 1.
Status of chimeric antigen receptor T-cell (CAR-T) approved by the Food and Drug Administration (FDA) or in Phase III clinical trials (as of July 1, 2019).
| Kymriah® (tisagenlecleucel-T; CTL019) | Yescarta® (axicabtagene ciloleucel; KTE-C19) | Lisocabtagene maraleucel (liso-cel, JCAR-017) | BB2121 | |
|---|---|---|---|---|
| Company | Novartis | Gilead (Kite) | Celgene | Celgene/Bluebird |
| Format | CARa-T | CAR-T | CAR-T | CAR-T |
| Co-stimulation | 4-1BB CS | CDf28 CS | 4-1BB CS | 4-1BB CS |
| Phase III name | B-ALLb: ELINA/DLBCLc: JULIET | TOWER | TRANSFORM | RRMM |
| Status | Approved by USFDA | Approved by USFDA | Phase III (NCT03575351) | Phase III (NCT03651128) |
| Cost (US) | $475,000 for B-ALL; $373,000 for R/Rd DLBCL | $373,000 | NAh | NA |
| Indication | B-ALL, R/R DLBCL | R/R DLBCL; PMBCLg | R/R DLBCL; CLLi | MMj |
| Sourced T-cell | Patient PBMCse; autologous; unspecified | Patient PBMCs; autologous; unspecified | Patient CD4 and CD8 T cells 1:1 ratio; autologous | Patient PBMCs; autologous |
| Vector | Lentivirus | Retrovirus | Lentivirus | Lentivirus |
| Patient group (=no.) | B-ALL = 63, R/R DLBCL = 93 | 101 | 73* | 33* |
| ORR | B-ALL = ND, DLBCL = 52% | 83% | 80%* | 85%* |
| CR | B-ALL = 83%, DLBCL = 40% | 58% | 59%* | 45%* |
| PR | B-ALL = 20%, DLBCL = 12% | 25% | 21%* | 39% |
| Median response duration time (months) | B-ALL = NR, DLBCL = 11.7 months | 11.1 months | 10.2 months* | 11.8 months* |
a
CAR, chimeric antigen receptor.
b
B-ALL, B-cell acute lymphoblastic leukemia.
c
DLBCL, diffuse large B-cell lymphoma.
d
R/R, relapsed or refractory.
e
PBMC, peripheral blood mononuclear cells.
f
CD, cluster of differentiation.
g
PMBCL, primary mediastinal large B-cell lymphoma.
h
NA, not applicable.
i
CLL, chronic lymphocytic leukemia.
j
MM, multiple myeloma.
*
Data from the Phase I trial result.