Contact: Ellen Bank
843.792.2626
May 13, 2004
Results of a preliminary study in this week’s issue of The Lancet suggest
that statins (cholesterol-lowering drugs) have potential in the treatment of
multiple sclerosis (MS).
Current drug treatments for MS are partially effective and expensive. Recent
knowledge that statins promote an anti-inflammatory response from the immune
system suggests a potential in the treatment of MS.
Inderjit Singh, Ph.D., scientific director of the Children’s Research
Institute of the Medical University of South Carolina (MUSC), and colleagues
report that 30 people with MS given 80 mg simvastatin daily for 6 months had
a 44% reduction in the proportion of brain lesions after three months of treatment
compared with lesions identified before treatment initiation.
“These findings suggest that an 80 mg daily dose of oral simvastatin over
a 6 month period could inhibit the inflammatory components of multiple sclerosis
that lead to neuro-logical disability,” said Singh. “Our results,
combined with the published work on the immunological effects of statins lend
support to the case for randomized controlled clinical trials to establish the
safety and efficacy of statins in the treatment of relapsing-remitting multiple
sclerosis.”
In an accompanying commentary Chris Polman from VU Medical Centre, Amsterdam,
Netherlands, concludes: “…[this] study is a big step forward because
it is the first to provide some evidence of an effect with a statin in multiple
sclerosis—but it is only an initial step. Additional data are required
to more precisely determine the clinical effects of statins, to explore the
optimum dose, the therapeutic window, and the differential potency of statins,
and to evaluate whether combination therapy might be more effective than monotherapy.
Physicians, scientists, companies, and regulatory agencies should now work together
to design and do randomized studies that have adequate power to address these
and other important issues. It is the joint responsibility of all involved to
ensure that some of the potential charms of statins (low-hurdle access, convenience,
low cost) do not develop into a dangerous boomerang, in case proper studies
become jeopardized by widespread off-label use.”
“The primary marker we measured was the change in the number of active
brain lesions associated with multiple sclerosis using MRI scans of the trial
participants at intervals during the trial,” said Lyndon Key, M.D., chairman
of the Pediatrics Department at MUSC, who designed the clinical trial with Singh,
William Tyor, M.D., and Timothy Vollmer, M.D.
Of the 28 participants who completed the trial, three showed no change in mean
number of lesions between pre- and post-treatment brain MRIs; two showed an
increase in lesions, and 23 showed a decrease in lesions.
“Not only were the results positive, but we used a drug that is used safely
by millions of people,” Key said. “The drug can be taken orally,
rather than by injection like the drugs currently used for MS, and costs about
a fifth that of other MS medications.”
The multicenter trial was run at MUSC with William Tyor, M.D., director of the
MUSC Multiple Sclerosis Clinical, serving as the clinical principal investigator.
Timothy Vollmer, M.D., at Yale University served as the clinical principal investigator
and lead clinical clinician for the study. Vollmer is currently at the Barrow
Neurological Institute in Phoenix. John Corboy, M.D., at the University of Colorado’s
Denver Health Sciences Center was a clinical principal investigator, and Valerie
Durkalski, Ph.D. at MUSC was lead biostatistician.
Singh and Key applied for, and received, an unrestricted educational grant from Merck and Co., Inc. to conduct the study. This trial was done with a limited number of participants, and all the participants in the trial received the active drug. The next steps in the research require trials with much larger numbers of participants with some on the drug and some not. To optimize the therapy, future trials could assess the safety of the combination of statins administered with other effective MS therapies.
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