New Ideas Fuel Next Generation Gene Therapy Research

Armen Hareyan's picture

Cancer Therapy

The concept seems straightforward. If, at its heart, cancer is a disease of genes, then giving patients new genes should disarm cancer. Such treatment would replace missing or faulty genes that keep cell growth in check, or would flush the body with "super genes" that could attack and destroy cancer. But as yet no such "gene therapy" for cancer has ever been approved by the United States Food and Drug Administration (FDA).

Past attempts to inject therapeutic genes directly into solid tumors have shown promise, but researchers have stumbled over efforts to treat multiple tumors or cancer that has spread. That's because a patient's immune system reacts against the therapy, or the genes can't find their way to cancer cells.


Albert Deisseroth, M.D., one of the first gene therapy researchers at The University of Texas M. D. Anderson Cancer Center, has said of numerous early attempts, "Our challenge to design gene therapy has been many times more complicated than finding the proverbial needle in a haystack."

Still, new and ambitious approaches to gene therapy at M. D. Anderson are seen as encouraging to many of the researchers involved. For example, a gene therapy strategy pioneered at M. D. Anderson is now on "fast track" approval at the FDA, the only gene therapy ever to be considered for approval for use in patients.

It has helped a number of lung cancer patients, including one who, at more than five years after treatment, is now the world's longest surviving gene therapy patient. And, at the opposite end of the spectrum, M. D. Anderson is conducting the world's first gene therapy trial aimed at preventing cancer. Patients at risk of developing oral cancer gargle a concoction twice a week that coats their throats with protective genes.

Researchers are testing a variety of novel approaches