BC-819, a plasmid comprising the H19 gene regulatory sequences and diphtheria toxin A, for the potential targeted therapy of cancers.

摘要: BC-819 (DTA-H19), in development by BioCancell Therapeutics Inc, under license from the Hebrew University of Jerusalem, is a double-stranded DNA plasmid carrying gene for A subunit diphtheria toxin regulation H19 promoter. H19, paternally imprinted, oncofetal gene, encodes an RNA that acts as riboregulator. Expressed at substantial levels embryonic and malignant tissues, but minimally or not expressed adult elevated expression has been observed over 30 malignancies prompting investigation into its utility targeted therapeutic agent. While most vivo studies have investigated treatment bladder cancer, recent also yielded encouraging results NSCLC,colon, pancreatic ovarian cancers. phase I/IIa clinical trial patients with non-muscle invasive cancer receiving intravesical reported mild local toxicity complete partial response rates 22 44%, respectively. At time publication, IIb was ongoing while I/II trials were accruing participants. This review provides focused summary existing experimental evidence demonstrating effectiveness construct, early outcomes discussion potential role therapy.