September 02, 2010

RNA Interference Success Versus Cancer

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Popular Science reports that biotech company Alnylam announced in June that its drug ALN-VSP cut off blood flow to 62 percent of liver-cancer tumors in those 19 patients, by triggering a rarely used defense mechanism in the body to silence cancerous genes. Whereas conventional drugs stop disease-causing proteins, ALN-VSP uses RNA interference (RNAi) therapy to stop cells from making proteins in the first place, a tactic that could work for just about any disease. “Imagine that your kitchen floods,” says biochemist and Alnylam CEO John Maraganore. “Today’s medicines mop it up. RNAi technology turns off the faucet.”

Novel Lipid Nanoparticles enabled systematic RNAi with one times more potency.

The Speculist has coverage

Alnylam Pharmaceuticals, Inc. made a total of 16 oral presentations at the American Chemical Society (ACS) Fall 2010 240th National Meeting & Exposition in Boston from August 22-26, 2010

Notable highlights from their presentations include results showing superior properties of canonical siRNAs compared with so-called 'dicer substrate' constructs, the application of 'click chemistry' approaches to the synthesis of siRNA conjugates, and the synthesis of novel cationic lipids for systemic delivery of siRNAs with lipid nanoparticles
There is a 12 webpage RNAi primer

Mammals’ immune systems made RNAi’s antiviral function irrelevant (although all vertebrates, including humans, still use RNAi to regulate mRNA activity), but researchers found that introducing small segments of double-stranded RNA to cells could trigger the ancient mechanism and selectively halt the production of specific proteins.

That ability makes RNAi a potential fix for many diseases, including cancer, that arise when abnormal cells produce excessive amounts of everyday proteins. In theory, manipulating RNAi to kill proteins is simple. ALN-VSP, for example, consists of synthetic double-stranded RNA designed to match tumor mRNA that codes for two proteins: VEGF, which cancers overproduce to help grow new blood vessels, and KSP, which sets off rapid cell division. The researchers send the synthetic RNA into liver cells, and the body’s RNAi system kills both the synthetic RNA and any matching tumor-grown mRNA. Knock out the mRNAs coding for those proteins—which in the liver are produced only by cancer cells—and the tumor stops growing.

“We can turn off any one of 20,000 genes with RNAi,” says Bruce Sullenger, a molecular biologist researching RNAi at Duke University. “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.

Alnylam 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. Alnylam is already testing RNAi therapy for Huntington’s disease and high cholesterol in cell cultures; other researchers are tackling macular degeneration, muscular dystrophy and HIV.

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the publication of new research findings in the journal Nature describing the discovery and validation of the role of the gene Sort1 in the development of cardiovascular disease, including myocardial infarction (MI)

A 44 page presentation on RNAi delivery progress.

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