Scientists are hopeful a major UK drug trial will establish that statins can be used to treat multiple sclerosis, reports The Guardian. The low-cost drugs are typically prescribed to help lower levels of “bad cholesterol” associated with raised risk of a heart attack or stroke, but they have also shown “incredible promise” for the treatment of MS.
A small study – involving 140 people – published in in 2014 on patients with secondary progressive MS, found those taking high doses of simvastatin had a significant reduction in the rate of brain atrophy over two years and also had better disability scores at the end of study.
The report says the new trial will be much larger, involving 1,180 people, and will aim to establish whether the drug can slow disability progression. The £6m project has funding from the National Institute for Health Research, the charities MS Society UK and National MS Society (US), as well as from the NHS and British universities.
Dr Jeremy Chataway, of the University College London Institute of Neurology, who led the 2014 trial, will also be at the helm for the new study. He said: “This drug (simvastatin) holds incredible promise for the thousands of people living with secondary progressive MS in the UK, and around the world, who currently have few options for treatments that have an effect on disability.
“This study will establish definitively whether simvastatin is able to slow the rate of disability progression over a three-year period, and we are very hopeful it will.”
More than 100,000 people in the UK have MS, which attacks the central nervous system. Symptoms usually start when a person is in their 20s and 30s and it affects almost three times as many women as men. It can be painful and exhausting and can cause problems with walking, moving, thought, memory and emotions.
The report says most of those diagnosed have relapsing MS and about 65% of these cases develop secondary progressive MS, usually within 15 years. There are currently no licensed treatments that can slow or stop disability progression in people with secondary progressive MS.
Michelle Mitchell, CEO of the MS Society, said the research would offer “a huge amount of hope”. She added: “This is a momentous step forward in our quest to find an effective treatment for progressive MS.”
Simvastatin is the most commonly used of the anti-cholesterol drugs. In the 2014 trial, patients were given 80mg doses, which would cost about 7p a day.
Stuart Nixon, who lives with secondary progressive MS, said in the report: “At the moment people like me are living with the prospect of our condition getting worse each day. This is the most exciting opportunity to change how we manage progressive MS. It would be amazing if this trial can show that an existing drug, costing just a few pence a day, can help with MS.”
Background: Secondary progressive multiple sclerosis, for which no satisfactory treatment presently exists, accounts for most of the disability in patients with multiple sclerosis. Simvastatin, which is widely used for treatment of vascular disease, with its excellent safety profile, has immunomodulatory and neuroprotective properties that could make it an appealing candidate drug for patients with secondary progressive multiple sclerosis.
Methods: We undertook a double-blind, controlled trial between Jan 28, 2008, and Nov 4, 2011, at three neuroscience centres in the UK. Patients aged 18–65 years with secondary progressive multiple sclerosis were randomly assigned (1:1), by a centralised web-based service with a block size of eight, to receive either 80 mg of simvastatin or placebo. Patients, treating physicians, and outcome assessors were masked to treatment allocation. The primary outcome was the annualised rate of whole-brain atrophy measured from serial volumetric MRI. Analyses were by intention to treat and per protocol. This trial is registered with ClinicalTrials.gov, number NCT00647348.
Findings: 140 participants were randomly assigned to receive either simvastatin (n=70) or placebo (n=70). The mean annualised atrophy rate was significantly lower in patients in the simvastatin group (0·288% per year [SD 0·521]) than in those in the placebo group (0·584% per year [0·498]). The adjusted difference in atrophy rate between groups was −0·254% per year (95% CI −0·422 to −0·087; p=0·003); a 43% reduction in annualised rate. Simvastatin was well tolerated, with no differences between the placebo and simvastatin groups in proportions of participants who had serious adverse events (14 [20%] vs nine [13%]).
Interpretation: High-dose simvastatin reduced the annualised rate of whole-brain atrophy compared with placebo, and was well tolerated and safe. These results support the advancement of this treatment to phase 3 testing.
Jeremy Chataway, Nadine Schuerer, Ali Alsanousi, Dennis Chan, David MacManus, Kelvin Hunter, Val Anderson, Charles R M Bangham, Shona Clegg, Casper Nielsen, Nick C Fox, David Wilkie, Jennifer M Nicholas, Virginia L Calder, John Greenwood, Chris Frost, Richard Nicholas