Tiny fat blobs are used to move protein-killing RNA into sick cells
Medical scientists have great power in their laboratories, and perhaps nowhere more so than with a technique known as RNA interference
By harnessing the potential of short bits of RNA inside cells, researchers have gained the power to shut off a gene’s capability to make proteins. Emerging in the last decade, this ability to “silence” genes holds great promise for treating human diseases by stopping the production of harmful proteins.
The tiny bits of RNA work well in cells, but getting them into human cells within the body has proved more problematic. That’s because the body’s defense mechanisms perceive the RNA bits as invaders and kill them before they can reach their intended target.
Now, a team led by Dr. Anil Sood, an associate professor at the University of Texas M.D. Anderson Cancer Center, may have solved the problem for ovarian cancer, opening the door to promising treatments for other forms of cancer.
Although there is no certainty those results will hold true in humans, the research represents a novel mechanism for transporting bits of RNA inside the body to desirable targets. The work was published earlier this month as the lead article in the journal Clinical Cancer Research.
Now, the research group is studying the RNA/liposome drug’s toxicity in human blood. So far, the results have been encouraging, with it appearing to cause no harm to blood cells and platelets.
Sood said he is in discussion with the U.S. Food and Drug Administration to begin formal toxicity studies within the next year, and he hopes to start human clinical trials in less than two years.
The therapy also has potential beyond ovarian cancer. The protein targeted by Sood’s drug is produced in excessive amounts by colon, breast, thyroid, and head and neck cancers.