A group of investigators at CalTech say they have successfully developed nanopolymers wrapped in a protein that can penetrate a cancer cell and deliver a small interfering RNA. And according to the report published in Nature, this is the first time that an RNAi therapy has been shown to work in humans.
The siRNA from Calando Pharmaceuticals is designed to block the cancer growth protein ribonucleotide reductase. And the researchers say that it worked as expected in people with melanoma.
"This directly interferes with the genetic mechanisms that promote cancer to stop the production of a particular protein," said Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society. "This is one step away from getting into the actual DNA."
"This is the first study to be able to go in there and show it's doing its mechanism of action," CalTech Professor Mark Davis told Reuters. "We're excited about it because there is a lot of skepticism whenever any new technology comes in." Davis worked as a consultant to Calando.
A number of biopharma companies have been working on delivering RNA therapies, including Alnylam, Roche and Pfizer, which announced a deal just days ago to develop a delivery technology with Tekmira that relies on fats, or lipids.