Statins for the treatment of multiple sclerosis: cautious hope.

cytokine profiles. These results provide hope, although they should be interpreted cautiously. The number of patients and the design of the study do not allow for a definitive conclusion on the role of statins in multiple sclerosis. The main concern with Vollmer and colleagues’ study is whether, without a placebo group, the reduction in disease activity as measured with MRI could be due to regression to the mean. The fact that patients were included in the study on the basis of the presence of gadolinium-enhancement might have selected for patients with active disease and who would have subsequently had reduced disease activity anyhow. Additional questions relate to other factors that might have had an effect on the trial’s outcome—eg, steroid use and the way any such use was accounted for, and any potential for unblinding of the readers of the MRI scans. Vollmer and colleagues did not find their exploratory immunological data to be supportive, which might be explained by the limited number of samples tested or might challenge the notion that meaningful antiinflammatory effects can be achieved at a daily dose of “only” 80 mg simvastatin. Vollmer and colleagues’ 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, drug companies, and regulatory agencies should now work together to design and do randomised 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 jeopardised by widespread off-label use.