New way to lower cholesterol

Scientists have lowered cholesterol in monkeys using a pioneering genetic technique that could provide a completely new way of treating human disease.

The ground-breaking study is the first demonstration of the approach, known as microRNA silencing, in non-human primates. Initial safety trials are now planned involving healthy human volunteers.

MicroRNAs (miRNAs) are short strands of RNA - a genetic molecule similar to DNA - that help regulate the way genes work. They are thought to play an important role in conditions ranging from viral infections and heart disease to neurological disorders and cancer.

Scientists in the US and Denmark used injections of a particular molecule to block, or "silence", liver miRNAs which control cholesterol metabolism.

Their findings were reported in the journal Nature. Applying the treatment to African green monkeys led to a dose-dependent lowering of blood cholesterol. Three shots of the drug was sufficient to achieve efficient silencing of the target microRNA, known as miR-22.

The Danish pharmaceutical company leading the research now intends to try out the same anti-miRNA compound on humans.

Copenhagen-based Santaris Pharma has patented the Locked Nucleic Acid (LNA) technology used to silence miRNAs by binding them to complementary molecules.

A Phase I clinical trial examining the safety of the drug is expected to get under way this year.

Professor Sakari Kauppinen, from the University of Copenhagen, who is director of microRNA research at the pharmaceutical company Santaris Pharma, said: "In this study, we used a simple intravenous delivery of.. LNA-antimR to antagonise (block) the liver-expressed microRNA-22 in African green monkeys, which resulted in long-lasting, efficient and reversible decreases in total plasma cholesterol without any evidence of adverse reactions.

"Even though further studies will be needed to optimise the dosing regimen and to assess the safety of LNA-antimiR compounds after long-term treatment, our findings represent an important step towards the development of LNA-based microRNA therapeutics."

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