Table 1:
Selected completed and future CAR T-cell therapy studies for pediatric solid tumors or brain tumors
| Disease | Target | CAR signaling domain | Vector | Cell type & delivery | Lymph depl | Comment, NCT #, or ref if published |
|---|---|---|---|---|---|---|
| Neuroblastoma | CD171 | ζ | pl | ATC, iv | − | 16 |
| CD171 | 41BB.ζ or CD28.41BB.ζ | LV | ATC, iv | + | NCT02311621 | |
| GD2 | ζ | RV | VST or ATC, iv | − | 17,18 | |
| GD2 | CD28.Ox40.ζ | RV | ATC, iv | +/− | pembrolizumab, 19 | |
| GD2 | ζ, ND | RV | ATC, iv | + | NCT03373097 | |
| GD2 | ζ, ND | RV | ATC, iv | +/− | NCT02761915 | |
| GD2 | ζ, ND | RV | iNKT, iv | + | 2nd transgene: IL15, NCT03294954 | |
| Neuroblastoma & sarcoma | GD2 | CD28.OX40.ζ | RV | ATC, iv | + | NCT02107963 |
| Sarcoma | HER2 | CD28.ζ | RV | ATC, iv | +/− | 21,22 |
| GD2 | CD28.OX40.ζ | RV | VST, iv | +/− | VZV Vaccine, NCT02932956 | |
| Hepatoblastoma & sarcoma | GPC3 | 41BB.ζ | RV | ATC, iv | + | NCT02932956 |
| Brain tumors | HER2 | CD28.ζ | RV | VST, iv | − | 24 |
| IL13Rα2 | 41BB.ζ | LV | ATC, ic | − | NCT02208362, 26 |
pl: plasmid, LV: lentivirus, RV: retrovirus, ATC: activated T cells, VST: virus-specific T cells, iNKT: invariant NK T cells, iv: intravenous, ic: intracranial, ND: not disclosed