Commonly Used Lipid-Lowering Drug Could Help Prevent Brain Atrophy in MS

Multiple sclerosis is a major cause of disability, particularly in young adults in temperate climates. Despite much success with drugs that substantially reduce relapse frequency during the initial inflammatory, relapsing-remitting phase, more than half of patients eventually develop non-relapsing, secondary progressive multiple sclerosis one to two decades after the onset of relapsing-remitting multiple sclerosis.

This chronic neurological deficit and increased brain atrophy are thought to be driven by neuroaxonal loss. 

Different medications such as immunomodulatory drugs have not proven effective when extended into secondary progressive sclerosis, and other direct neuroprotective strategies have also failed. 

Commonly Used Drug May Have Another Application

3-hydroxy-3-methylglutaryl-CoA (HMG-CoA) reductase inhibitors (statins) are extensively used to treat primary hyperlipidemia and for the prevention of myocardial and cerebral ischemia. 

Initial studies with early-stage multiple sclerosis showed that high-dose (80 mg) simvastatin significantly decreased lesion activity. 

A phase 2 trial by Chataway and colleagues published in the journal The Lancet showed that a high dose of simvastatin reduced the annualized rate of whole-brain atrophy in patients with MS. 

For the study, the team included a total of 140 participants that were randomly assigned to receive simvastatin or a placebo and found a 43% reduction in the annualized rate in the treatment group when compared to placebo. No major adverse events were reported during the duration of the study. 

The team is already doing a phase 3 testing to evaluate the effectiveness of the therapy in a larger treatment group. 


Chataway J, et al. Effect of high-dose simvastatin on brain atrophy and disability in secondary progressive multiple sclerosis (MS-STAT): a randomised, placebo-controlled, phase 2 trial. Lancet. 2014 Jun 28;383(9936):2213-21. doi: 10.1016/S0140-6736(13)62242-4. Epub 2014 Mar 19. PMID: 24655729. 

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