THURSDAY, Oct. 3 (HealthDay News) -- An experimental drug that
lowers LDL "bad" cholesterol by helping sweep it from the
bloodstream appears to be both safe and effective in its first
The drug known as ALN-PCS reduced cholesterol an average of 40
percent in the small, early study, and, if proven to work in large
trials, potentially could replace or complement statins, the
Currently, statin drugs such as Lipitor, Crestor and Zocor are
widely used to control cholesterol. One heart doctor not involved
with the new study said another class of drugs might be useful.
"Cardiovascular disease remains the leading cause of death of men and women globally and reduction of LDL cholesterol with statin medications has been demonstrated to substantially reduce the risk of first or recurrent cardiovascular events," said Dr. Gregg Fonarow, a professor of cardiology at the University of California, Los Angeles.
However, while statin therapy is very effective for heart risk
reduction and generally well tolerated, a substantial number of
people cannot achieve ideal LDL cholesterol levels despite high
statin dosing, he said. And others can't take statins at all.
"As such there is an important need to develop addition therapies to lower LDL cholesterol," Fonarow said.
The new trial demonstrated dramatic reductions in LDL
cholesterol on top of statin therapy, he said, and "these agents
are being further evaluated in very large randomized controlled
clinical outcome trials."
The report was published Oct. 3 in the online edition of
In this early, phase I trial, researchers tried the drug on 32
healthy patients with mild to moderate raised low-density
lipoprotein (LDL) cholesterol. Patients were randomly assigned to
one of six doses of ALN-PCS or an inactive placebo.
The drug, which is given intravenously, significantly reduced
cholesterol. Those given the highest dose saw an average 40 percent
reduction in LDL cholesterol, the researchers found.
ALN-PCS was well tolerated, and a similar number of patients in
both groups had mild to moderate side effects (79 percent compared
to 88 percent), such as a temporary rash. In addition, the drug
didn't cause any significant changes in liver function or
inflammation, the researchers said.
The investigational drug works by blocking the production of a
protein called PCSK9 that regulates cholesterol. This protein
destroys LDL receptors that clear the artery-clogging cholesterol
from the blood, the researchers explained.
Prior studies have shown that gene mutations that cause an
increase in PCSK9 lead to increased LDL cholesterol, while gene
mutations causing less PCSK9 appear to lower cholesterol. In
addition, statins may actually boost levels of PCSK9, which could
reduce their effectiveness, the researchers noted.
RNA interference is a process of gene "silencing" using small
molecules of RNA to shut off particular genes -- for example,
"The next step will be larger, multi-dose studies to address the long-term safety and tolerability of ALN-PCS in various patient populations, including those on statins and those who are statin-intolerant," said study co-author Kevin Fitzgerald, senior director for research at Alnylam Pharmaceuticals, which helped develop the drug and funded the new study.
"Statins work for some patients but not for everyone," he added, and new medicines are needed "to lower LDL-cholesterol and reduce the risk of coronary artery disease."
To find out more about cholesterol, visit the
American Heart Association.
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