BC-819 is BioCancell’s leading product, commencing pivotal (Phase III) bladder cancer clinical trials in 2016. BC-819 is a DNA plasmid that does not contain inflammatory or viral elements. It is composed of two regions of interest, the H19 promoter and the Diphtheria Toxin A sequence.
The H19 gene is expressed only in cancerous cells, activated by an array of H19-specific transcription factors exclusive to these cells. When BC-819 enters a cancerous cell, the Diphtheria Toxin ‘A’ chain (DTA) is synthesized under the regulation of H19, and the cell dies. The synthesized DTA is unable to infect surrounding cells. When BC-819 enters a healthy cell lacking the H19-specific transcription factors, the plasmid remains inactive and is cleared from the body. In this way, BC-819 serves as a “search and destroy” unit, where cells expressing the H19 gene and exposed to the plasmid trigger their own demise, while healthy cells are not affected.