TUESDAY, March 18, 2014 (HealthDay News) -- High doses of the cholesterol-lowering drug simvastatin -- sold under the brand name Zocor -- appeared to slow brain shrinkage in patients with multiple sclerosis, according to a small, early study from England.
In patients with the secondary progressive (chronic) stage of multiple sclerosis, brain shrinkage was reduced 43 percent for those taking Zocor compared to patients taking placebos, the researchers said.
"This effect is provisional and requires a larger phase 3 study, but holds promise for all types of MS," said Dr. Jacqueline Palace, a consultant neurologist with Oxford University Hospitals and co-author of an accompanying journal editorial.
"Because it is a repurposed drug and already has a good safety profile and is cheap, it could become available fairly quickly if further studies confirm the suggested effect," Palace said.
The report was published in the March 19 online edition of the journal The Lancet.
Zocor is part of a class of drugs known as statins, which are commonly prescribed for patients with high cholesterol.
Although how Zocor works to reduce brain shrinkage isn't known, Palace speculated that the drug might protect the brain by targeting inflammation.
However, Dr. Emmanuelle Waubant, a professor of clinical neurology and pediatrics at the University of California, San Francisco, questioned whether the reduction in brain shrinkage was due to Zocor or some other factors.
In her own research with MS patients using another cholesterol-lowering drug, Lipitor, Waubant did see a reduction in the development of brain lesions, something that these researchers didn't see. "That is very surprising," she said.
In addition, among Waubant's patients, there was no reduction in brain shrinkage after taking the drug for a year, but there was a reduction among the patients in this current study, something she also finds surprising.
Waubant said these findings are promising, but MS patients shouldn't start taking these drugs in hopes of slowing the progression of their condition.
"This was a small study," she said. "Before we can be certain there is a positive effect of this medication on the progression of MS, we need to reproduce that in other studies, because sometimes findings are a fluke."
Moreover, if clinical symptoms aren't improved with treatment, it makes the use of the drug problematic, Waubant said.
"It's one thing if you slow down progression of brain atrophy, but if it doesn't translate into improvement in clinical outcomes for patients, it may not be useful," she said. "If it's real, that would be great."
For the new phase 2 study, a team lead by Dr. Jeremy Chataway, who at the time of the study was at Imperial College London, randomly assigned 140 MS patients to receive either 80 milligrams of Zocor a day or a placebo.
When the researchers compared MRIs taken at the start of the trial with those taken two years later, they found that patients taking Zocor showed a 0.3 percent overall reduction in the rate of brain shrinkage each year.
"Normally brain shrinkage occurs in progressive MS at about 0.6 percent per year, and high-dose Zocor reduced that over two years by about 43 percent," Chataway said.
Chataway, now a consultant neurologist at University College London Hospital, said he believes Zocor might be protective of brain tissue or circulation in the brain.
To be of real benefit to patients, Zocor has to have an effect on the progression of disability, not just brain shrinkage, Chataway said. "We need to move on to phase 3 trials to show it has a clear effect on disability," he said.
"This may be the first step toward treatment in secondary progressive MS for which there is no treatment. It's the first step, but a very exciting step," Chataway said. "But I don't want everyone to go out there and start Zocor."
In addition to reducing brain shrinkage, there were modest improvements in clinical symptoms as rated by doctors and reported by patients, the study found.
In the early stages of MS, patients experience intermittent symptoms, called relapsing-remitting MS.
Within 10 to 15 years, more than half of patients develop secondary progressive MS. This involves a steady worsening of symptoms and an increase in disability. No drugs have shown a positive effect in this chronic stage of the disease, the researchers said.
Simvastatin, the generic form of Zocor, costs about $5 to $10 for 30 pills. It's covered by most insurance plans, including Medicare.
Perhaps slowing brain shrinkage will also slow the progression of disability, said Dr. Karen Blitz, director of the North Shore-LIJ Multiple Sclerosis Center, in East Meadow, N.Y. "That's key," she said. "That's what we need to find out."
Blitz said other cholesterol-lowering drugs might work as well as Zocor. However, she isn't advising patients to start taking Zocor to slow the progression of MS before a bigger phase 3 trial is done.
"Patients of mine who have high cholesterol should probably consider taking Zocor now because there could be an added benefit," she said.
To learn more about multiple sclerosis, visit the U.S. National Library of Medicine.