- An autologous cell vaccine demonstrated improved OS and PFS with low toxicity among patients with glioblastoma (GBM).
- Survival was higher among patients with tumors bearing methylated O6-methylguanine-DNA methyltransferase (MGMT).
Why this matters
- GBM is uniformly fatal, with a 14-month median survival and few treatment options.
- Phase 1b study of 33 patients with newly diagnosed GBM randomly assigned to autologous cell vaccine for 1 of 4 exposure periods; randomization was halted after patient 23, and subsequent patients received highest exposure.
- Vaccine was a combination of GBM cells and an antisense molecule against insulin-like growth factor type 1 receptor DNA/mRNA.
- Exposure categories (chambers/hours implanted):
- 10/24 (6 patients)
- 10/48 (5 patients)
- 20/24 (5 patients)
- 20/48 (17 patients)
- Median (range) follow-up: 13 (4-39) months.
- Funding: Paparone Foundation, Stevens Foundation, Kimmel Cancer Center.
- 2-year OS was 34% and 1-year PFS was 41% (vs 14% and 28% with standard of care).
- Post-hoc analysis demonstrated 16 patients with methylated- and 17 with non-methylated-MGMT tumors; OS was higher in the methylated (30.9 months) compared with the unmethylated (11.3 months) group independent of temozolomide treatment.
- 12 of 17 progression-free patients are alive and functioning well.
- Small study size.
- No control arm.