Table 7.
Key clinical trials assessing immunotherapy for cancer patients with brain metastases.
| Tumor Type | Phase | Drugs (Target) |
n | Trial arm | Intracranial RR (%) | Median PFS | Median OS | Trial No. (Ref.) |
|---|---|---|---|---|---|---|---|---|
| Melanoma | I | Ipilimumab (CTLA-4) | 17 | Ipilimumab + SRS/WBRT | 65 | 2.5 mos | 8.0 mos | NCT 01703507 [45] |
| Melanoma | I | Nivolumab (PD-1) | 17 | Nivolumab + SRS | 60 | / | / | NCT 02716948 [51] |
| Melanoma | II | Ipilimumab (CTLA-4) | 72 | (1) Asymptomatic: Ipilimumab; (2) Symptomatic: Ipilimumab |
(1) 25 (2) 10 |
(1) 1.9 mos (2) 1.2 mos |
(1) 7.0 mos (2) 3.7 mos |
NCT 00623766 [40] |
| Melanoma | II | Nivolumab Ipilimumab (PD-1/CTLA-4) | 90 | Nivolumab + Ipilimumab induction followed by Nivolumab maintenance | 57 | 59% at 9 mos | 82% at 9 mos | NCT 02320058 [35] |
| Melanoma | II | Ipilimumab (CTLA-4) | 86 | Ipilimumab + Fotemustine | 35 | 3.0 mos | 12.7 mos | NCT 01654692 [33,34] |
| Melanoma | II | Nivolumab Ipilimumab (PD-1/CTLA-4) | 76 | (1) Asymptomatic: Nivolumab + Ipilimumab; (2) Asymptomatic: Nivolumab; (3) Symptomatic: Nivolumab |
(1) 44 (2) 20 (3) 6 |
(1) 50% (2) 29% (3) 0% at 6 mos |
(1) 75% (2) 59% (3) 44% at 6 mos |
NCT 02374242 [21] |
| Melanoma/NSCLC | II | Pembrolizumab (PD-1) | 65 | Pembrolizumab | 22 33 | 2.0 mos | 17.0 mos | NCT 02085070 [18] |
| NSCLC/RCC | II | Nivolumab (PD-1) | 26 | Nivolumab + SRS | 42 | 6.1 mos | 21.4 mos | NCT 02978404 [52] |
| Breast Cancer |
I | Nivolumab (PD-1) | 14 | Nivolumab + SRS | 55 | / | / | NCT 03807765 [53] |
| Melanoma | II | Nivolumab Ipilimumab (PD-1/CTLA-4) | 128 a | Nivolumab + Ipilimumab followed by Nivolumab or Nivolumab + SRS | / | / | / | NCT03340129 [54] |
| NSCLC | III | Pembrolizumab (PD-1) | 108 | Pembrolizumab + Chemotherapy | / | / | / | NCT 02578680 [55] |
| NSCLC | III | Atezolizumab (PD-L1) | 124 | Atezolizumab | / | / | 16.0 mos | NCT 02008227 [56] |
| Solid Tumors |
II | Pembrolizumab (PD-1) |
101 | Pembrolizumab ± SRS | 40 | / | / | NCT 02886585 [39] |
a, Estimated Enrollment; mos, months; NSCLC, non-small-cell lung cancer; RCC, renal cell carcinoma; RR, response rate; PFS, progression-free survival; OS, overall survival; SRS, stereotactic radiosurgery; WBRT, whole brain radiotherapy.