HOUSTON—An experimental gene therapy targeting pancreatic cancer reduced or eradicated tumors, inhibited metastasis, and prolonged survival in experiments in two mouse models, and did so with very limited toxicity. Researchers at M.D. Anderson Cancer Center are compiling additional preclinical data for FDA review and hope to begin a phase I trial of the therapy in 1 to 2 years.
"This vehicle, or vector, is so targeted and robust in its cancer-specific expression that it can be used for therapy and perhaps for imaging," said senior author Mien-Chie Hung, PhD, professor and chair of the Department of Molecular and Cellular Oncology. He and his colleagues reported their preclinical findings in Cancer Cell (12:52-65, 2007).
"There are no good options for pancreatic cancer patients now," said James Abbruzzese, MD, professor and chair of M.D. Anderson's Department of Gastrointestinal Oncology, who with other clinicians is now working with Dr. Hung to bring the therapy to clinical trials. "This looks like a promising approach to gene therapy for pancreatic cancer."
Dr. Hung and his team call the system a versatile expression vector and nicknamed it VISA. The molecularly engineered therapy specifically embeds a mutated, more lethal version of the Bik gene—which expresses a protein that signals cancer cells to destroy themselves—into pancreatic cells.
The system consists of a targeting molecule, or promoter; two components that enhance gene expression in the target tissue; and the mutated gene, which the researchers have designated BikDD. These components are packaged in liposomes and delivered intravenously.
M.D. Anderson has licensed the technology to Alchemgen Therapeutics Inc., a Houston-based company cofounded by Dr. Hung.
In the preclinical studies, the researchers tested VISA against two aggressive pancreatic cancer lines in two different mouse models.
