Researchers from University College London in the UK, have conducted a phase II trial of a type of statin called simvastatin, in people with secondary progressive MS. The results have been published today in the prestigious medical journal The Lancet.
Phase II trials are an intermediate step in the clinical trials process, designed primarily to test safety and obtain an early indication of whether a drug may be an effective treatment.
In this trial 70 people with secondary progressive MS received a high dose of 80mg simvastatin per day for two years, while a control group of 70 people with secondary progressive MS received a ‘dummy pill’ (placebo).
Over the two years of the trial, people receiving simvastatin had a slower rate of brain tissue loss (atrophy) compared to those who received the placebo. The rate of brain atrophy in those people taking the placebo was 0.58% per year. This is consistent with the rate of tissue loss seen in untreated people with secondary progressive MS observed in other studies. The people receiving simvastatin tissue loss of 0.28% per year, equating to a 43% reduction in brain atrophy.
The extent of whole brain atrophy has been shown in other studies to correlate with disability progression. While the effects were small, disability progression was slowed in the people who received simvastatin compared to those on placebo. This was true for both physical and cognitive disability measurements. In this trial, simvastatin did not have any effect on relapses or brain lesions as seen by MRI scans.
The safety profile of simvastatin is already well established from its use for the treatment of high cholesterol, however the dose used in this trial in MS was double the maximum dose used for cholesterol control. Despite this, the trial results showed no difference in the number of adverse or serious adverse events experienced by participants in the simvastatin and placebo arms, indicating that this dose appears to be safe in people with secondary progressive MS.
The treatment of secondary progressive MS is currently a significant unmet need for people with MS. This trial is an encouraging development, but it will be crucial to see that this effect on brain atrophy and safety can be replicated in a much larger phase III trial and can be shown to correlate with a clinically meaningful reduction in physical and cognitive disability.
View a summary of the Lancet paper here.