The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MabVax Therapeutics’ vaccine for the treatment of relapsed or recurrent high-risk neuroblastoma in remission or with limited residual disease after best available treatment. The bivalent vaccine is intended to elicit a targeted immune response against the two most common antigens on neuroblastoma cells, GD2 and GD3.
Results of a recent phase I trial published in Clinical Cancer Research showed encouraging outcomes with the vaccine in a small cohort of patients with relapsed neuroblastoma.1 Antibody responses against GD2 and/or GD3 were seen in 12 of 15 patients, and disappearance of minimal residual disease was documented in 6 of 10 patients assessable for response. ■
1. Kushner BH, Cheung IY, Modak S, et al: Phase I trial of a bivalent gangliosides vaccine in combination with β-glucan for high-risk neuroblastoma in second or later remission. Clin Cancer Res 20:1375-1382, 2014.