Potential Heart Disease Breakthrough

Nov. 4, 2003 -- An amazing new treatment promises to unclog arteries -- even in people with advanced heart disease.

It's called ApoA-I Milano. The original molecule was found in the blood of an Italian family with unusually healthy arteries -- despite high levels of fats in their blood and low levels of protective "good" HDL cholesterol. Now a genetically engineered version of this "good" HDL cholesterol protein has been tested in a small human trial.

The results -- published in the Nov. 5 issue of the Journal of the American Medical Association --astounded even the doctors who performed the trial. Steven E. Nissen, MD, medical director of The Cleveland Clinic's cardiovascular coordinating center, gave five weekly injections of ApoA-1 Milano to patients with acute heart disease caused by plaque- clogged arteries.

"We really didn't think it was going to work," Nissen tells WebMD. "Nobody was more shocked than I was when the statisticians handed me the data. ... It is unprecedented. Nobody has ever seen this kind of plaque regression. It really is an epiphany."

Surprise -- and Vindication

The findings exceed even the most optimistic expectations, says Daniel J. Rader, MD, director of preventive cardiology at the University of Pennsylvania School of Medicine in Philadelphia. Rader's editorial comment on the Nissen study appears in the same issue of JAMA.

"This is, frankly, very surprising. I haven't talked to a single person in the field who isn't surprised," Rader tells WebMD. "And the speed with which the change occurred is remarkable. Nobody ever expected you could impact plaque in that short a time. This opens door to treating atherosclerosis in a more acute way to try to stabilize and regress plaque in patients with acute heart disease. This paper suggests it's no longer theory but reality."

But one person isn't surprised. That's Prediman K. (PK) Shah, MD, director the atherosclerosis research center at Cedars-Sinai Medical Center, Los Angeles, and professor of medicine at UCLA.

Shah has been studying ApoA-I Milano for nine years. He's the first major researcher to argue in favor of the once-laughed-at theory behind the new treatment. His animal studies led directly to the new treatment, dubbed ETC-216 by Esperion Therapeutics Inc., Ann Arbor, Mich. Shah serves as a consultant to the company.

"We've seen this in animal studies over the last eight years: Plaque regression in five weeks, with positive changes in 48 hours," Shah says. "For us, it was not a surprise. The unprecedented nature of the human study is the rapid plaque regression in just five weeks. It's never been seen before."

How It Works

Everybody has heard that there are two kinds of cholesterol. There's the "bad" LDL cholesterol, which clogs the walls of your arteries when there's too much of it. And there's the "good" HDL cholesterol, which removes excess cholesterol from the body and takes it to the liver for elimination.