PRE-CLINICAL AND CLINICAL VALIDATION OF AN ANTI-CANCER MODALITY THAT ABLATES REFRACTORY, LOW GROWTH FRACTION TUMORS

Intratumoral expression of the E. coli purine nucleoside phosphorylase (PNP) gene was originally described by our laboratories as a means to inhibit growth of solid tumors in vivo. The approach generates purine bases that disrupt DNA, RNA, and protein synthesis, a unique mechanism when compared with all approved or experimental cancer therapeutics. Use of PNP has been validated by numerous laboratories worldwide against human tumor xenografts (lung, liver, pancreas, bladder, glioma, and prostate, among others). Data from a recently completed phase 1 clinical trial has indicated substantial anti-cancer activity in human subjects with no serious toxicities.