AFTVac

These findings establish AFTV as a promising treatment option for GBM and highlight the importance of molecular profiling in treatment selection. Future studies should explore combining AFTV with immune checkpoint inhibitors to enhance efficacy in PD-L1-positive cases.

Negative immunostaining for PIK3R2 as well as negative p53 revealed an increased survival in patients receiving AFTV therapy for GBM. In patients receiving AFTV, these immunostaining results may serve as a predictor of treatment efficacy and overall survival.

Given the growing interest in immunotherapy (IMT), we developed an autologous formalin-fixed tumor vaccine (AFTV) manufactured from the patient's own glioblastoma multiforme (GBM) tissue in paraffin-embedded blocks made from the resected tumor and a double-blind, randomized phase IIB trial of AFTV with temozolomide in newly diagnosed GBM was conducted. The 3-year progression-free survival (PFS) rate for patients with gross total resection (GTR) on imaging tended toward improvement: 81% in the AFTV group versus 46% in the placebo group (P = .067). Based on these IIB results, the feasibility of conducting a phase III trial was confirmed for IIB-eligible patients with total resection. We here plan to conduct the world's first double-blind, randomized, placebo-controlled phase III trial using Cellm-001 to demonstrate autologous tumor immunostimulant efficacy. This IMT, in combination with sub-analyses (GTR, P53 status, CD8 score, and other factors) to be validated, is expected to be a breakthrough in effective standards of care for the treatment of GBM.

A p53-negative status in IHC and GTR were the predictive factors for AFTV treatment in newly diagnosed GBM patients. Microenvironment-targeted treatment and pretreatment blood cell status may be key factors to enhance therapy effects.