Experimental Medications: Other Therapeutic Strategies
Statins are oral medications that are most commonly prescribed to lower cholesterol. Current interest is based on a non-controlled observational study (a study without a placebo group), suggesting that the risk of developing new brain lesions was reduced by about half in people with early forms of MS who were taking Lipitor® (atorvastatin). However, a three-year Danish study of individuals with RRMS failed to find any beneficial effect for simvastatin as an add-on therapy to Avonex. The use of statins to lower cholesterol in people taking interferons should be discussed with a healthcare professional to consider the potential benefits versus risks.
Chataway and colleagues published the results of the MS-STAT trial in 2014. This Phase II study evaluated whether high-dose simvastatin could slow the rate of whole-brain atrophy, disability, or both, in 140 patients with SPMS. The trial showed that daily treatment with 80 mg of simvastatin was associated with a statistically significant benefit compared to placebo on the Expanded Disability Status Scale (EDSS) at two years. The rate of brain atrophy also was decreased in people receiving simvastatin.
Researchers then evaluated how simvastatin treatment affected other aspects of SPMS. These secondary analyses showed that the statin had a positive effect on the functioning of the brain’s frontal lobe and on health-related quality of life, but did not show a benefit relative to placebo in terms of preserving or slowing the rate of decline of verbal and non-verbal memory.58
The initial results and subsequent analyses from the Phase II MS-STAT trial suggest the value of conducting larger studies of statins and their impact on clinical outcomes in SPMS. A Phase III trial, MS-STAT2, will further test the benefits of simvastatin for people with SPMS. This is an exciting area for further inquiry because of the need for effective treatments for this form of the disease.