RNAi Therapy Proves Promising

By Rachel Howell Stockton

For many of us, the first introduction we had into the nebulous world of robotics was back in the days when it was relegated to sci-fi thrillers; the clearest image we had of the concept was of the gauche machine from Lost in Space – the one who repeatedly, week after week, announced, “Danger! Will Robinson!”

Nearly fifty years later, the reality of this particular type of technology has gone far beyond even Steven Spielberg’s imagination, it seems.

The journal Nature is reporting on new nanotechnology wherein a group of chemical engineers from Caltech have created robots to enter the blood stream and directly into cancerous tumors.  The technology is referred to as RNA interference (or RNAi), and is showing promising results.

RNA stands for ribonucleic acid, a chemical that scientists believe can disable proteins that are involved in many types of cancers, blindness and even AIDS.

According a Caltech press release issued March 21, researchers have known about the therapeutic properties RNA interference since 1998; however, until now, taking aim at the right target was problematic. Because of the physicality of the targeted genes, the proteins responsible for illness were often missed because they remained hidden.

For the current research, tiny “robots” were sent into the bloodstream to locate the cancer producing proteins inside a tumor; once inside, they breakdown, blocking the gene that produces the protein.

Reuters reports that in a phase 1 clinical trial involving melanoma patients, subjects were injected intravenously four times a day over a 21 day period.  After the trial, three patients were tested – it was then confirmed that the nano-particles had entered the tumors; the supposition is that the RNA therapy had done its job.

Certainly, there are other efficacy and safety studies that must be conducted; but for now, it appears that RNAi therapy has a promising future.