April 15, 2010 (Toronto) -- Statin drugs show promise for the treatment of multiple sclerosis (MS), a small, preliminary study suggests.
Statin drugs are best known for their cholesterol-lowering effects. But the drugs "have anti-inflammatory effects on immune cells as well, which is why we figured they could be beneficial in MS," says study leader Scott Zamvil, MD, PhD, associate professor of neurology at the University of California, San Francisco.
MS is an autoimmune disease in which the body's own immune cells mistakenly recognize cells in the brain and spinal cord as foreign and attack them. This leaves behind scars or lesions and disrupts the ability of nerves to transmit information, resulting in disability.
In the new study of people with early forms of multiple sclerosis, the statin drug Lipitor cut the risk of developing new brain lesions by about 50% compared with placebo. New lesions are reliable indicators of future MS attacks, according to the researchers.
The study was presented at the annual meeting of the American Academy of Neurology.
Lipitor Cuts Risk of Developing New MS Lesions
The researchers had intended to study more than 150 people who had their first MS attack, but enrollment was stopped due to slow recruitment after 81 patients entered the trial.
The study failed to meet its primary goal, which was to show that Lipitor could prevent a second attack or the development of three or more new brain lesions as viewed on MRI scans within a year.
That was "because we didn't have sufficient patients to show such an effect," Zamvil tells WebMD. "But on an important imaging measure, we were able to show a significant benefit," he says.
Specifically, the study showed that over a 12-month period, 55% of people given Lipitor had no new brain lesions compared with 28% of the placebo group.
Statins and MS: Where Do We Go From Here?
So should the research proceed to a larger study? Zamvil says that it would be difficult to embark on another study pitting statins against placebo, as several oral medications that attack MS at its roots have been shown to be effective since this study was undertaken.
But, says Nicholas G. LaRocca, PhD, it might be worthwhile to mount a study using statins in combination with the new MS drugs, known as cladribine and fingolimod. LaRocca, vice president for health care delivery and policy research at the National Multiple Sclerosis Society in New York City, was not involved with the study.
"We don't want to miss out on something beneficial just because the primary [goal] was not met," Zamvil says.
Statins have the benefit of being "very, very safe," he says. And once Lipitor's patent expires in 2011, it will also be inexpensive, he says.
Based on the current findings, no one should consider taking statins -- alone or in combo with other drugs -- as a treatment for MS, LaRocca and Zamvil stress.
Asked if all statin drugs have the same anti-inflammatory effects, Zamvil says that studies in the lab suggest that Lipitor and Crestor outperform other statins on this measure.
This study was sponsored by the National Institute of Allergy and Infectious Diseases with support from Biogen-Idec, the Nancy Davis Foundation, and Pfizer Pharmaceuticals, the maker of Lipitor.