Summary: Investigators at the University of California, San Francisco Multiple Sclerosis Center and at 13 other centers in North America are conducting a clinical trial evaluating the effects of oral Lipitor® (atorvastatin, Pfizer, Inc.) compared with inactive placebo in 152 people at high risk for developing MS. Scott Zamvil, MD, PhD, and Emmanuelle Waubant, MD, PhD, of UCSF, are leading this effort. The study is supported by the Immune Tolerance Network, a research consortium funded by the NIH's National Institute of Allergy and Infectious Diseases.
Rationale: Previous studies have suggested that cholesterol-lowering “statins” can alter immune responses in a way that may hold promise in treating MS. Drs. Zamvil and Sawsan Youssef (Stanford University) and others reported that Lipitor prevented or reversed the MS-like disease EAE in mice (Nature, November 7, 2002). In addition, a small open-label pilot study by Timothy Vollmer, MD, and colleagues in 28 people with relapsing-remitting MS found that the cholesterol-lowering pill Zocor® (simvastatin) safely reduced the number of new lesions (Lancet, May 15, 2004). The mechanism underlying the drug’s possible promise appears to be immune system modulation, rather than a cholesterol-lowering mechanism.
Eligibility and Details: People eligible for participation include individuals 18-55 years of age with no previous neurological history, who experience a clinically isolated syndrome (CIS, a single demyelinating event, for example, an attack of optic neuritis in one eye, or an episode of numbness on one side) lasting at least 48 hours and magnetic resonance imaging findings suggestive of MS. Patients must receive a three- to five-day course of corticosteroids starting within sixty days of onset of a CIS and must be enrolled within 90 days of onset of a CIS.
Participants will be randomly assigned to receive either Lipitor (80 mg/day) or placebo; 91 people will receive the treatment, and 61 will receive placebo for a treatment phase of 12 months. At the end of the treatment phase, all participants will be followed for an additional 6 months. If individual participants experience a clinical attack (exacerbation) or show significant MRI changes, they will be offered Avonex® (interferon beta-1a), a standard treatment for MS, for the remainder of their enrollment. Participants will undergo careful neurological and MRI monitoring to detect disease activity as early as possible.
The primary objective of the study is to evaluate the effects of Lipitor to decrease or delay clinical and MRI disease activity. Secondary objectives are to determine if Lipitor is safe in patients with CIS, to determine which immune system cells or molecules are modified by Lipitor, to evaluate the effects of Lipitor on brain tissue loss, and to determine potential residual benefits after the discontinuation of therapy.
Contact: A list of sites follows. Future updates will be available in the “Trials Recruiting Patients” section of the National MS Society Web site.
-- Research & Clinical Programs Department
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