Brachyury-targeting immunotherapy for chordomas: treatment with GI-6301, a Saccharomyces cerevisiae yeast-based cancer vaccine

Introduction: Chordomas are slow growing rare tumors deriving from notochordal remnants, occurring in locations along the neuro-axis from the skull base to the sacro-coccygeal spine. Treatment includes surgery, with en bloc resection when feasible, photon/proton based radiation therapy, and molecularly targeted agents. However overall long-term prognosis remains guarded. Areas covered: We review the role of GI-6301, a yeast-brachyury based therapeutic cancer vaccine for immnotherapeutic treatment of patients with chordomas, and review it’s pharmacology and clinical efficacy demonstrated in a completed Phase I trial. Expert opinion: Brachyury is highly expressed in chordomas. GI-6301 immunotherapy aims to provide a therapeutic option for patients with chordoma, particularly for those who have failed conventional surgery and irradiation, and for whom treatment options are limited. A Phase I dose-escalation trial has shown promising results. A phase II study with GI-6301 in patients with locally advanced chroma is underway.