"A new experimental cholesterolfighting drug is creating quite a buzz among researchers and other experts prompting some to use words such as dramatic breakthrough and game changing to describe earlystage clinical trial results. If these initial results pan out in larger trials the drug could prove to be more effective than statins the widely prescribed drugs which have been very successful in lowering levels of LDL  or  bad cholesterol http_UCM__Article.jsp. Researchers have known for some time that when the protein PCSK which stands for proprotein convertase subtilisinkexin  binds to LDL receptors on the liver it compromises the organs ability to filter the bad cholesterol from the blood.   Too much LDL cholesterol circulating in the blood can lead to the thickening of artery walls making them less flexible and therefore impairing their function and increasing the risk of heart disease. In this phase one clinical trial which is designed to determine if a drug is safe researchers found that using a monoclonal antibody labproduced protein called REGN was not only safe but effectively blocked PCSK and therefore signficantly reduced bad cholesterol in healthy patients as well as those also taking the popular cholesterollowering drug Lipitor. Earlier studies had shown this effect in primates but this new study published Wednesday in the New England Journal of Medicine http_home provides the first clinical trial data for testing the drug on humans. This new drug is being developed by Regeneron Pharmaceuticals and Sanofi who funded the research. Wars for PCSK are far bigger than the statin wars said Dr. Evan A Stein lead author of the study and researcher at the Metabolic and Atherosclerosis Research Center in Cincinnati Ohio. This is a hot research area and everybody is so close together. Other companies including Amgen Merck  Company Novartis and Pfizer are also hot on the trail developing their own PCSK inhibitor drugs. Stein said. Additional results for PCSK inhibitor drugs are expected to be presented at the annual American College of Cardiology meeting  httpaccscientificsession.cardiosource.orgACC.aspxwhich begins Saturday in Chicago he said. The REGN study included three trial arms. Two arms used  healthy volunteers who were either injected with a single dose of the drug in increasing amounts to test for side effects which is the purpose of a phase one clinical trial.  A third arm included  people with a family history of high cholesterol and  people with nonfamilial high cholesterol. All of those subjects were also receiving treatment with the statin Lipitor.   A control group of subjects with nonfamilial high cholesterol was treated only with a special diet.  None of the subjects who received REGN discontinued the study because of adverse effects and the subjects who received REGN had a striking reduction of  to   in LDL cholesterol according to Stein. Stein characterized the results of the trial as pretty dramatic. A PCSK inhibitor Stein said differs from statins because its unlike any other drug. With statins you get toxicity  with these drugs we dont see any side effects with the antibody. In an accompanying editorial authors Dr. Stephen G. Young and Loren G. Fong Ph.D. write At this point the status of PCSK therapeutics appears to be full speed ahead. Soon we can expect more human trials in which investigators will dissect the properties of different PCSK antibodies and assess the effect of these agents. However without longterm safety data and evidence that PCSK inhibitors truly help prevent heart disease Young and Fong caution that it will remain unclear how important this class of drugs will be. The cost of this drug will also play a role in determining which patients might use it Fong and Young say.  But they also note that patients who cannot tolerate statins could benefit greatly. If the results are confirmed This is game changing science said Dr. Richard A. Stein a spokesman for the American Heart Association whos not related to the study author or involved in the research. Stein called the trial results a very important breakthrough while being careful to note that they represent early science which should be confirmed through larger and longer studies. He said the study methodology was thorough because it included people with high cholesterol as well as people with genetic familial high cholesterol which is proven to be a result of impaired PCSK genetic function."