April 2, 2003 (Honolulu) — A six-month pilot study of the relatively inexpensive oral cholesterol-lowering drug simvastatin has been shown to reduce the number of lesions in multiple sclerosis (MS) by 43%.
Lead investigator Timothy Vollmer, MD, chairman of the division of neurology at Barrow Neurological Institute in Phoenix, Arizona, reported the results here at the American Academy of Neurology 55th annual meeting. Dr. Vollmer said the drug, given to 28 patients at 80 mg a day for the six-month study, also reduced the volume of inflammation and the number of new areas of inflammation.
The study, designed to test safety, did not detect any improvements in disability or number of new attacks.
"We don't yet know whether this has a benefit for decreasing their attack rate or disability," Dr. Vollmer said at a press briefing. But he added, "The patients tolerated it really well. No patient stopped due to side affects."
Two of the patients, he said, had mildly elevated liver tests, which resolved after stopping the drug.
He said the study suggested several advantages of statins over the five most commonly used MS drugs:
The three interferons, glatiramer acetate, and mitoxantrone cost $10,000 or more a year, compared with $1,000 to $2,000 a year for simvastatin.
The current drugs are by injection, daily or monthly, compared with oral administration of simvastatin.
Four of the drugs have what he termed "nuisance" adverse effects: pain and swelling at the site of injection and occasionally flu-like symptoms. Mitoxantrone, a chemotherapeutic agent, is associated with infections, secondary neoplasms, and organ damage to the heart and liver. Although it is more effective than the other drugs, its adverse effect profile limits it to 5% to 10% of the MS population.
The drugs are only partially effective, producing 30% fewer attacks and a modest slowing of progression.
Study patients were selected on the basis of having at least one gadolinium-enhancing lesion out of a series of three pretreatment monthly magnetic resonance imaging (MRI) scans. Patients were then treated for six months with MRIs obtained at months 4, 5, and 6. Primary outcome was change in number of enhancing lesions.
Dr Vollmer believes the drug works by suppressing the TH1 CD4 cells.
"Anything that suppresses that seems to be helpful," he said. Research on mice and rats has shown statins are effective at suppressing inflammatory cells from entering the brain.
Other research suggests that statins might be able to enter the brain and work directly on key target sites, suggesting a benefit for the secondary progressive phase of MS, for which, Dr. Vollmer added, other therapies have very little benefit.
Meanwhile, "the study indicates there may be a benefit for statins in MS in one way or another. But how it would be used has not been worked out," he said.
He is beginning preliminary planning that he hopes will lead to a trial in the next year or so on the use of one or more statins in combination with currently approved therapies.
Dr. Vollmer envisions sufficient results in about four years for a consensus to emerge about the role of statins for MS.
In a phone interview, Stephen Reingold, MD, vice president of the National Multiple Sclerosis Society in New York, who was not involved in the study, agreed that statins have advantages over some drugs traditionally used to treat MS.
But, he said, "it's a small study," and a clinical trial lasting at least a couple years is needed to see the desired outcomes in clinical management.
This study was supported by an unrestricted educational grant from Merck Inc.
AAN 55th Annual Meeting: Abstract S11.004. Presented March 31, 2003
Reviewed by Gary D. Vogin, MD
Medscape Medical News © 2003 Medscape
Cite this: Larry Schuster. Statin Appears Promising for MS - Medscape - Apr 02, 2003.