Table 1.
Diagnosis, prior therapy, product characteristics, and survival
| Study phase/ dose level | ID | Diagnosis | Prior therapies | Total dose level/ dose×109/kg |
Weight | Total dose (×109) | TX completed | Product | |||||||
| Viability (%) | Cytotoxicity (%) | Cd3 (%) | Cd8 (%) | Cd4 (%) | Response end of therapy | OS (days) | Status | ||||||||
| PI/1 | IT20107 | OST | CT: MAP, HD Ifosfamide | 0.32 | 60.0 | 19.04 | Yes | 73 | 25 | 92 | 57 | 40 | PD | 75 | DOD |
| PI/1 | IT20108 | OST | CT: MAP, IE | 0.32 | 55.0 | 17.76 | Yes | 75 | 10 | 88 | 57 | 38 | PD | 139 | DOD |
| PI/1 | IT20111 | NB | CT: SCT, Topo/ Cy, RT | 0.32 | 62.9 | 20.08 | Yes | 79 | 53 | 98 | 31 | 61 | SD | 1476 | DOD |
| PI/1 | IT20113 | NB | CT German NB 2004, SCT, multiple lines, sirolimus | 0.64 | 19.4 | NA | No | 45 | NA | NA | NA | NA | NA | 92 | DOD |
| PI/2 | IT20115 | OST | Multiple lines CT (MAP, IE, gemcitabine, docetaxel), RT | 0.64 | 66.9 | 42.8 | Yes | 74 | 19 | 99 | 49 | 51 | SD | 593 | DOD |
| PI/2 | IT20116 | DSRCT | CT, pazopanib | 0.64 | 67.4 | 43.1 | Yes | 92 | 33 | 98 | 64 | 37 | PD | 201 | DOD |
| PI/2 | IT20119 | NB | ANBL12P1, SCT, vaccine, m3F8, irino/TMZ | 0.64 | 29.3 | 18.75 | Yes | 77 | 3 | 96 | 6 | 93 | PD | 590 | DOD |
| PI/3 | IT00010 | NB | ANBL12P1, SCT, hu3F8, RT, vaccine | 1.28 | 18.0 | 23.04 | Yes | 91 | 31 | 95 | 84 | 22 | PD | 494 | DOD |
| PI/3 | IT20122 | NB | ANBL0532, SCT, RT, Ch14.18 | 1.28 | 25.0 | 32 | Yes | 86 | 21 | 83 | 31 | 42 | PD | 633 | DOD |
| PI/3 | IT00003 | DSRCT | CT, Surg, RT | 1.28 | 91.7 | 37.8 | No | 95.8 | 5 | 94 | 43 | 78 | PD | 83 | DOD |
| PI/3 | IT00005 | NB | ANBL12P1, MSK13-260(m3F8), CT, MSK12-230 (Hu3F8), RT, | 1.28 | 23.3 | 29.9 | Yes | 93.6 | 10 | 98 | 43 | 57 | SD | 1571 | AWD |
| PII | IT00013 | NB | ANBL0532, SCT, RT, Ch14.18, CT | 1.28 | 51.4 | 88.96 | Yes | 88.6 | 47.9 | 97 | 52 | 49 | PR | 1640 | NED* |
| PII | IT00017 | NB | ANBL12P1, SCT, RT, ANBL 0032 (ch14.18), CT, Hu3F8 | 1.28 | 75.8 | 78.40 | Yes | 93.8 | 17.4 | 94 | 50 | 47 | SD | 1405 | AWD |
| PII | IT00019 | NB | CT, Hu3F8, RT, SCT+Haplo, NK cells | 1.28 | 28.5 | 34.24 | Yes | 92.1 | 21.1 | 84 | 48 | 54 | SD | 935 | DOD |
| PII | IT00022 | NB | German NB 2004, MIBG, Hu3F8, Multiple CT | 1.28 | 37.6 | 48.00 | Yes | 88.4 | 68.1 | 87 | 48 | 46 | SD | 614 | DOD |
| PII | IT00023 | NB | ANBL12P1, Ch14.18, MIBG, Hu3F8, vaccine, Hu3F8, Donor NK | 1.28 | 25.2 | 25.60 | Yes | 83 | 45.7 | 97 | 45 | 44 | PD | 220 | DOD |
| PII | IT00028 | NB | CT, RT, Hu3F8, Vaccine, MIBG, | 1.28 | 32.6 | 41.60 | Yes | 94.1 | 47.9 | 83 | 46 | 52 | PD | 709 | DOD |
| PII | IT00031 | NB | ABNL0532, tandem SCT, RT, Ch14.18, irino/TMZ/Ch14.18 | 1.28 | 56.6 | 59.04 | Yes | 96.9 | 24.8 | 90 | 63 | 39 | PD | 1460 | AWD |
| PII | IT00033 | NB | ANBL12P1, SCT, RT, ANBL0032, Ch14.18; topo/cy | 1.28 | 39.2 | 50.16 | Yes | 86.5 | 35.1 | 96 | 86 | 19 | SD | 1218 | AWD |
| PII | IT00040 | NB | ANBL0532, tandem SCT, RT, Ch14.18 | 1.28 | 22.0 | 28.00 | Yes | 94.1 | 43.1 | 98 | 68 | 37 | SD | 902 | DOD |
| PII | IT00084 | NB | ANBL12P1, SCT, RT, ANBL1221 (irino/TMZ, Ch14.18) | 1.28 | 67.2 | 69.04 | Yes | 84.2 | 12.1 | 98 | 40 | 59 | PD | 439 | AWD |
*After disease progression at 6 months and then receiving several additional lines of therapy.
ANBL1221, COG NB protocol for recurrent /refractory disease; AWD, alive with disease; ch14.18, chimeric GD2 mAb; COG, Children’s Oncology Group; CT, chemotherapy; DOD, died of disease; DSRCT, desmoplastic small round cell tumor; GM-CSF, granulocyte macrophage colony-stimulating factor; hu3F8, humanized 3F8 GD2 mAb; IE, ifosfamide and etoposide; irino/TMZ, irinotecan and temozolomide; MAP, methotrexate, doxorubicin, cisplatin; m3F8, murine 3F8 GD2 mAb; NB, Neuroblastoma; OS, overall survival; OST, Osteosarcoma; PD, Progressive disease; PI, phase I; PII, phase II; RT, radiation therapy; SCT, autologous stem cell transplant; topo/cy, topotecan and cyclophosphamide.