Italians Save the Day

Breakthrough Mutant Cholesterol Repairs Heart Disease
By rickyjames, Section News Posted on Wed Nov 5th, 2003 at 07:23:33 AM PST
It's an obscure passage in an obscure document in an obscure U.S. Government database, but it's a true life X-File and X-Men comic rolled into one: "Analysis of the genealogic tree of the complete family groups showed that the apoprotein (apo) AIMilano is transmitted as an autosomal dominant trait, all carriers coming from a single mating couple, living in the eighteenth century." One of those blissful young lovers was a mutant. Today their offspring live in the idyllic Italian town of Limone sul Garda, and they all have a mutant form of cholesterol running through their veins that prevents them from having the atherosclerosis or fat-clogged arteries that is the cause of death for a sobering one out of every five fatalities in America every year. Now this mutant form of cholesterol has been synthesized and given to a group of heart attack patients in a controlled scientific study. The results have just been announced, and they are amazing. Update [2003-11-5 8:3:17 by rickyjames]:: You know, it occurs to me that the ironic yet logical end result of this APO gene research would be french fries that are good for you...

 

As reported in USA Today and a host of other news sources: Taking their cue from nature, researchers have developed the first treatment shown to dramatically shrink the fatty plaques that clog arteries, a study reports Wednesday. The experimental drug is a synthetic version of a protein discovered in a handful of Italian villagers who had healthy arteries and lived long lives despite having low levels of good cholesterol, called HDL. In its place, the 40 villagers have a different form of HDL, a protein dubbed ApoA-1 Milano. The new research shows that a genetically engineered version of the protein reversed the progression of coronary artery disease, says lead researcher Steven Nissen of the Cleveland Clinic. "The concept is sort of liquid Drano for the coronary arteries," said Nissen, a cardiologist. Five infusions of ApoA-1 over six weeks reduced plaque deposits by an average of 4%, 10 times the reduction seen after years of taking cholesterol-lowering statin drugs. The benefit was even more pronounced in the large, inflamed plaque-filled bulges that are prone to burst and cause heart attacks, reducing them by as much as 34%. "I almost fell out of my chair when I saw what happened," Nissen says. Although the study was small, involving just 47 patients with severe heart disease, the results were so dramatic that researchers say it heralds the start of a new offensive against heart disease. Currently, researchers concentrate on drugs that lower levels of bad cholesterol (LDL), which carries fats into arteries. In the future, researchers will try bolstering HDL, which hauls fats away. The treatment must be given intravenously, so it's likely to be used in people who have had heart attacks, says the study in The Journal of the American Medical Association. "The ApoA-1 story is remarkable, beginning with discovery of rare individuals in a small Italian town and leading to the development of a potential new therapy," Daniel Rader of the University of Pennsylvania says in an accompanying editorial. Esperion, the biotech firm that makes the protein, is planning a much larger trial involving 1,500 to 2,000 patients, prior to seeking government approval to market ApoA-1. Government approval is probably a decade away. Back