In time, targeted gene therapy may help improve the outlook for many types of cancer. While we have a long way to go, I am increasingly optimistic about new gene therapy approaches to earlier treatment and, ultimately, to a strategy that may help prevent cancer development. |
It all started here at M. D. Anderson with the concept, vector development, preclinical trials and the first phase of testing. This agent has clinical benefit, and because of its novelty and lack of interest by major pharmaceutical companies, the only way to make it available to patients was to start a biotech company. But it has been a long road. |
P-53 has been called the guardian of the genome for the cell. |
The healthy P-53 gene can then begin to trigger the self-destruction program for the cancer cell. |
This is a first step in showing that we can restore a normal, functioning gene in a cancer cell, a gene that normally causes that cell to be cancerous when it's defective. |