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An experimental drug doctors hoped would be the next big advance in preventing heart attacks and strokes failed a crucial test, researchers reported Saturday.
The medicine, which reduces levels of a particle in the blood associated with heart attacks and strokes, didn't protect volunteers in a closely watched study.
More from the original report
"I was shocked. I had high hopes for success," says Steven Nissen , a cardiologist at the Cleveland Clinic who led the study. "Cardiovascular disease is still the leading cause of death in developed countries. And so we really were hoping to find additional therapies that would reduce risk."
The research aimed to help people who seem to do everything right. They keep their cholesterol and blood pressure low, eat well, maintain a healthy weight, and exercise regularly. But many still suffer heart attacks and strokes.
"That sort of patient we see very frequently, and we were really doing our best to try to find a way to help those patients," Nissen says.
Many of these patients have elevated blood levels of a cholesterol-carrying protein called lipoprotein(a) or Lp(a), which has long been thought to increase the risk for heart disease.
"About 20% of the global population have elevated levels. And that's about a billion people. So it's a common disorder," Nissen says.
Until recently, doctors didn't have any drugs that could lower Lp(a) levels. But several drug companies have developed experimental new medications in recent years that can slash elevated Lp(a).
So Nissen and his colleagues gave 8,350 adults who already had heart disease either a placebo or a drug called pelacarsen, which cuts Lp(a).
The drug did suppress Lp(a) levels by about 78%, the researchers showed. But after five years, that drop didn't translate into saving people from suffering cardiovascular complications.
"Very disappointing, terribly disappointing," says Nissen, who presented the first details about the study at an American Heart Association meeting in Chicago Saturday and published more specifics in the New England Journal of Medicine and the NEJM Evidence .
Drugmaker Novartis, which sponsored the study,…