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Drug Insight: using statins to treat neuroinflammatory disease


Statins, a family of 3-hydroxy-3-methylglutaryl coenzyme A reductase inhibitors, are used primarily to reduce atherogenesis and cardiovascular morbidity. Surprisingly, they have also been shown to have immunomodulatory properties that might be of benefit for the treatment of autoimmune disorders. Statins can prevent and even reverse ongoing paralysis in experimental autoimmune encephalomyelitis—the mouse model for multiple sclerosis—and on the basis of these findings, statins are now being tested in patients with multiple sclerosis in clinical trials.

Key Points

  • Statins are cholesterol-lowering drugs with immunomodulatory properties that might be of benefit in the treatment of neuroinflammatory disorders such as multiple sclerosis (MS)

  • Most of the currently known statin-mediated immunomodulatory effects seem to be related to the inhibition of HMG-CoA reductase, a key enzyme in the cholesterol biosynthetic pathway

  • Studies in mice with experimental autoimmune encephalomyelitis have shown that statins interfere with multiple aspects of immune cell function

  • At present, there is insufficient epidemiological evidence to support the use of statins to treat MS in humans, but the initial results of clinical trials have been encouraging

  • Statins are also being tested in other inflammatory conditions, including rheumatoid arthritis and Alzheimer's disease

  • Although statins are perceived as safe and well-tolerated drugs, they have side effects that should be considered, particularly when they are administered in combination with other agents

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Figure 1: Cholesterol biosynthesis and isoprenylation of Ras and Rho.


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Correspondence to Scott S Zamvil.

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Weber, M., Prod'homme, T., Steinman, L. et al. Drug Insight: using statins to treat neuroinflammatory disease. Nat Rev Neurol 1, 106–112 (2005).

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