Molecular Engineering of “Pathotropic” Targeting for Cancer Gene Therapy

by Jennifer Noe Pahre, JD, Michael Fuchs, JD, John P. Levy, Erlinda M. Gordon, MD, and Frederick L. Hall, PhD
Volume 3, Issue 1 (January/February 2004)

More than 70 percent of all prospective gene transfer/gene therapy protocols are designed to treat metastatic cancer. A large number of such protocols involve strategies to attempt cancer immunization via cell-based gene transfer of cytokines or tumor antigens, while others involve the delivery of oncolytic viruses or vectors bearing prodrugs, chemoprotective agents, antisense constructs, or tumor suppressor genes. However, a major unresolved problem that has impeded the progress of cancer gene therapy to the clinic is that of inefficient gene delivery to target cells in vivo. In this regard, the advent of pathotropic targeting launches a new paradigm in cancer gene therapy. By targeting the histopathology of the cancerous lesion — rather than the cancer cells per se — to effectively concentrate the gene vector within primary and metastatic tumors, the safety and efficacy of intravenously administered vector nanoparticles were increased significantly in animal models of cancer. This article describes the development of the pathotropic Targeted Delivery System (TDS) that now serves as the guidance system for “smart” nanoparticles bearing designer killer genes for cancer gene therapy…

Noe Pahre J, Fuchs M, Levy JP, Gordon EM, Hall FL. Molecular Engineering of “Pathotropic” Targeting for Cancer Gene Therapy. BioProcess J, 2004; 3(1): 21-26.