Statins like Lipitor and its generics have revolutionized cardiovascular care for nearly two decades as an effective, inexpensive way to reduce LDL cholesterol, the so-called bad cholesterol in the bloodstream.
Not everyone can take them, though; a significant number of people complain of muscle pain, weakness and cramping so severe that they discontinue the therapy even at the risk of a heart attack or stroke. Their resistance to the medication has been controversial, because in most cases there are no biomarkers for the muscle problems individuals describe. Some researchers have speculated that the problem is psychological, the "nocebo" effect of blaming the medication for the pain.
But a study released Sunday shows for the first time that statin intolerance is very real and that a newer, different kind of drug can have a strong impact on these patients' high cholesterol.
Researchers found that 42.6 percent of people who had complained of muscle pain while taking at least two different statins experienced the same symptoms when given a statin during the study but had no ill effects when administered a placebo. They were then able to reduce their LDL cholesterol levels by more than half when given a PCSK9 inhibitor, evolocumab, for 24 weeks, compared with just a 16.7 percent reduction on ezetimibe, another medication.
The Food and Drug Administration approved PCSK9 inhibitors last year, but only for small groups of people, including those with an inherited disease that dramatically elevates their LDL cholesterol levels.
"This problem of statin intolerance is one of the most vexing problems for both patients and physicians in cardiovascular medicine," Steven E. Nissen, chairman of the Department of Cardiovascular Medicine at the Cleveland Clinic, said in an interview. He said the new study, which he led, offers hope for people who have not been able to take statins because of side effects.
The research was unveiled Sunday at the American College of Cardiology's annual conference and published simultaneously in the medical journal JAMA. It was funded by Amgen, maker of evolocumab, an injectable PCSK9 inhibitor. Nissen has worked with many pharmaceutical companies to determine the efficacy of heart therapies but requires the companies to donate any payments to charity so that he receives no compensation or tax breaks, according to a bio accompanying the study.
Heart disease is the leading killer of Americans, and some 73 million people in the United States have high LDL cholesterol, which can greatly increase their cardiovascular risk. Millions of people take statins to lower their risk. In 2013, the American College of Cardiology and the American Heart Association released new treatment guidelines that would substantially increase the number of people on statins.