AUBURN, Maine -- An experimental medicine shows great promise in treating a condition that causes sky-high cholesterol levels among some French Canadians, according to a Maine cardiologist involved in the hot new area of pharmaceutical research.
The genetic disorder, called familial hypercholesterolemia or FH, can cause exceedingly high levels of artery-clogging cholesterol, even in children. The condition is more common in people of Native American or French Canadian descent, and has cropped up in the Lewiston-Auburn area, which is home to many Franco Americans.
Over the last year, roughly 1,000 people participating in clinical trials for the medicine have seen their LDL cholesterol, or "bad cholesterol," drop significantly with no side effects, said Dr. Robert Weiss, a cardiologist at Auburn-based Maine Research Associates, which is conducting clinical studies on the medicine and has overseen a genetic study on FH.
The research participants took the medication, part of a group of medicines known as "PCSK9 inhibitors," along with cholesterol-lowering drugs called statins. The medication could eventually help not only individuals diagnosed with inherited FH, but millions of other people who can't lower their cholesterol sufficiently with statins alone, Weiss said.
"I've been doing research on drugs for 30 years," he said. "This could be perhaps the second most important thing that we've ever worked on, with the statins themselves being the first one. If you can lower bad cholesterol into the 40s and 50s and stop heart disease and stroke, the benefit to people and to society as a whole, both medically and financially, is really breathtaking."
Four pharmaceutical companies are researching the medication, which still needs federal approval, he said. Given by injection, the drug is designed to "knock out" an enzyme in the body that prevents the liver from removing bad cholesterol from the bloodstream, Weiss said.
"It's an enzyme in the body that breaks down a piece of the mechanism that eats up cholesterol," he said. "If you don't have enough of that, you get very high cholesterol."
Studies are now being conducted to prove the medication can lower cholesterol enough to prevent heart problems, a key step to gaining U.S. Food and Drug Administration approval, he said. That greenlight could take a couple of years, he said.
Patients would self-administer the drug by shot once a week or once a month, Weiss said. Most would take it along with a statin, such as Lipitor, an affordable class of drugs that works well for many people, he said.
The medication could particularly benefit people with FH, whose bodies struggle to clear cholesterol from the blood and for whom statins often aren't enough, he said.
"It's a naturally occurring substance, everybody has some of this [PCSK9 inhibitor in the body]," Weiss said. "But some people need more than others because of this genetic issue."
The medication, which is expected to be costly, could also benefit people who are intolerant to statins, he said.
In Maine, half of people with heart disease live with cholesterol levels outside a healthy target range, Weiss said. Some may skip their pills or may need their drug regimen adjusted, but for many people, statins aren't enough to attain a healthy cholesterol level, Weiss said. If this medication can change that, there's good reason to hope that it could reduce heart attacks and strokes, he said.
Maine Research Associates is looking for volunteers to participate in its clinical study of the medication. Sixteen participants have already signed up, but more volunteers are needed, Weiss said. For information, call Maine Research Associates at 782-9835. ___