Are statins a treatment option for multiple sclerosis?

Background Treatment options for multiple sclerosis (MS) are limited. The immunomodulatory drugs interferon beta and glatiramer acetate are only partly effective in reducing the relapse rate, slowing disease progression, and diminishing the number and volume of lesions on MRI. Mitoxantrone is an immunosuppressant approved for the treatment of active forms of relapsing-remitting or secondary progressive MS, but is dose-limited owing to its potential cardiotoxicity. Thus, identifying new effective therapeutic options with few side-effects is highly desirable. Recent developments Evidence has emerged that statins, which are inhibitors of 3-hydroxy-3-methylglutaryl coenzyme A reductase, have immunomodulatory effects. Recent reports showed that statins prevent and reverse chronic and relapsing experimental autoimmune encephalomyelitis, an animal model of MS. Furthermore, in vitro experiments with human immune cells have shown an immunomodulatory profile of statins comparable to that of interferon beta. An open label clinical trial of simvastatin for MS revealed a significant decrease in the number and volume of new MRI lesions and a favourable safety profile. Where next? The obvious advantage of statins over existing MS therapies is their oral route of dosing. Statins might be beneficial for MS patients as monotherapy or as an add-on to established disease modifying drugs. As the evidence of the benefit of statins in MS is currently insufficient, large controlled clinical trials are needed. The first of these trials is about to start.