Targeted RNAi Versus Liver Cancer
An example of the next generation of targeted cancer therapies: "Since last April, 19 cancer patients whose liver tumors hadn't responded to chemotherapy have taken an experimental drug. Within weeks of the first dose, it appeared to work, by preventing tumors from making proteins they need to survive. The results are preliminary yet encouraging. With a slight redesign, the drug might work for hundreds of diseases, fulfilling the promise that wonder cures like stem cells and gene therapy have failed to deliver. ... We can turn off any one of 20,000 genes with RNAi. The challenge has been to get a drug into only the desired cells and not harm others. ... Researchers have worried that a drug might disrupt normal protein production in a healthy cell, or that the immune system will destroy the drug before it reaches its target. [Scientists] overcame both concerns by packaging the drug in a fatty envelope that is absorbed primarily by the liver. This allowed doctors to administer the drug through the blood, rather than by an injection to one spot, which improves results by ensuring that the entire liver receives an even dose. The technique's ability to attack single genes could lead to drugs for the 75 percent of cancer genes that lack any specific treatment, as well as for other illnesses. [Researchers are] already testing RNAi therapy for Huntington's disease and high cholesterol in cell cultures; other researchers are tackling macular degeneration, muscular dystrophy and HIV. The potential has driven nearly every major pharmaceutical company to start an RNAi program."
Link: http://www.popsci.com/science/article/2010-07/rx-every-disease