April 2, 2003 (Honolulu) -- Researchers have presented the first human findings looking at a cholesterol-lowering drug for multiple sclerosis treatment. They found that Zocor not only reduces the size but also the number of brain lesions from MS.
Animal research touting cholesterol-lowering drugs called "statins" as a multiple sclerosis treatment hit the scene just last year, and the research has quickly moved from mice to humans. In a recent animal study, researchers found that Lipitor -- another statin -- prevented disease progression and reversed paralysis in mice with a multiple sclerosis-like disease.
In the current study, Timothy Vollmer, MD, and colleagues studied 28 people with the most common form of multiple sclerosis, called relapsing-remitting -- in which periods of relapse, when symptoms flare up, alternate with periods of remission, when symptoms fade. Vollmer, chairman of the division of neurology at the Barrow Neurological Institute in Phoenix, presented the results in Honolulu at the annual conference of the American Academy of Neurology.
The study participants -- aged 18 to 55 -- had evidence of at least one brain lesion from multiple sclerosis on an MRI brain scan. Each participant then took 80 mg of Zocor daily and underwent a series of MRI brain scans during the study. The study was supported by Merck Inc., Zocor's manufacturer.
Six months into treatment, the researchers found that the number of brain lesions was reduced by 43%. In addition, the researchers projected that Zocor would decrease the annual rate of relapses from 43% before treatment to 32% after treatment.
Zocor as a multiple sclerosis treatment was well tolerated by the patients, Vollmer says. "No patient stopped due to side effects."
When used to lower cholesterol, Zocor and other statins can occasionally raise liver enzymes -- indicating that the drug may be harming the liver. In this study, two of the patients had mildly elevated liver tests, which resolved after stopping the drug, says Vollmer.
Vollmer says he believes Zocor and other statins may work by suppressing an overactive part of the immune system.
"Anything that suppresses seems to be helpful," he says, and research on mice and rats has shown statins to be effective at suppressing inflammatory cells from entering the brain.
One possible additional benefit of the drug, Vollmer says, is that it appears that Zocor might be able to enter the brain to work directly on key target sites. If so, that raises the possibility that Zocor may be an effective multiple sclerosis treatment for slowing progression of the disease, Vollmer says.
But even though the study indicates that there may be a role for statins in multiple sclerosis treatment one way or another, exactly how they would be used still has to be worked out, Vollmer says.
He is beginning preliminary planning for a trial in the next year or so on the use of one or more statins in combination with currently approved multiple sclerosis treatments.
Vollmer envisions that there ought to be enough research completed in about four years for a consensus to emerge about statins' potential role as multiple sclerosis treatments.
Stephen Reingold, PhD, vice president of research for the National Multiple Sclerosis Society in New York, tells WebMD the advantages of the drug are that it's oral, it's well known, it's not expensive, and side effects are low.
"It's a small study," and it needs a clinical trial lasting at least a couple of years to see how well statins work for multiple sclerosis treatment.
Vollmer says that statins -- if proven to be of benefit in future studies -- could have several advantages over current multiple sclerosis treatments.
- Commonly used multiple sclerosis treatments, such as interferon, Copaxone, and Novantrone, cost $10,000 or more a year, compared with $1,000 to $2,000 a year for Zocor.
- Current multiple sclerosis treatments are by injection, compared with statins, which are taken orally.
- Four of the current multiple sclerosis treatments have what Vollmer termed nuisance side effects: pain and swelling at the site of injection, and occasionally flu-like symptoms.
- Novantrone, a chemotherapy drug, has been associated with infections, cancer, and heart and liver damage. Though it's often more effective than other multiple sclerosis treatments, its side effects limits it to 5% to 10% of people with multiple sclerosis.