Cell Encapsulation Technology (CET) allows the standardized release of GM-CSF by genetically modified allogeneic cells loaded within a capsule. We present the final results of the Phase I clinical trial assessing this patient specific, cell-based therapy, combining the implantation of irradiated autologous tumor cells and capsules containing allogeneic cells genetically engineered to produce huGM-CSF.
For this single center, first in human Phase 1 trial, 15 pts with progressing, solid tumors refractory to all standard treatments were enrolled. Immunizations was performed in healthy skin, distant from tumor deposits. Patients were treated with 6 sc immunizations (week 1-2-3-4-6-8) combining 4x106 irradiated autologous tumor cells and 2 capsules containing 8x105 MVX-1 cells, producing >20ng/24h of huGM-CSF. Capsules were removed after 8 days and analyzed for huGM-CSF production. Primary endpoints are safety and feasibility. Secondary endpoints include clinical outcome and immunomonitroing.
15 patients are available for safety and feasibility. Tt is very well tolerated with only one G3 AE related to IMP. Main toxicity is G1-2 minor discomfort during caspules sc implantation. No treatment related SAE were reported. Clinical grade therapeutic products were successfully manufactured for all patients. 95% % of explanted macrocapsules showed good ex-vivo huGM-CSF production. >50% of patients (8/15) experienced either PR or SD including disappearance of lung metastasis, prolong survival. Correlation between positive DTH and OS was observed. Immunomonitoring by Elispot shows increased INFg production by PBMC after immunization.
MVX-ONCO-1 is the first personalized cell-based immunotherapy combining autologous tumor cells and sustained local production of GM-CSF at the vaccination site using cell encapsulation technology. This technology has a very good safety profile safe with no systemic SAE related to therapy. In this heavily pretreated population, clinical activity (partial response and stable disease ) was observed in >50 of patients.
Clinical trial identification
Legal entity responsible for the study
Geneva University Hospital, Unité de recherche clinique de la Fondation Dr Henri Dubois-Ferrière Dinu Lipatti
N. Mach: I am the co-founder of MaxiVAX SA, I am the inventor of MVX-ONCO-1 I am a minority shareholder of MaxiVAX SA. All other authors have declared no conflicts of interest.