Cholesterol-lowering drugs do not work against multiple sclerosis

Results from the world's first Phase 3 trial of secondary progressive multiple sclerosis (MS), led by UCL researchers, have shown that the commonly used cholesterol-lowering drug simvastatin cannot slow the progression of disability.

The results, announced at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS 2024) conference in Copenhagen, followed promising findings from Phase 2 trials.

However, the researchers are convinced that the MS-STAT2 study, which was a collaboration between British universities (including UCL), the MS Society, the National Institute for Health Research (NIHR), the National MS Society (USA) and the NHS, nevertheless represented a huge milestone in MS research.

MS-STAT2 is the largest study to date on secondary progressive MS, enrolling nearly 1,000 participants for whom there are currently limited treatment options.

Multiple sclerosis is a lifelong disease that affects the central nervous system (brain and spinal cord). The main feature of secondary progressive MS is that symptoms gradually worsen and accumulate over time, which is called progression.

Lead investigator of the MS-STAT2 trial, Professor Jeremy Chataway (UCL Queen Square Institute of Neurology), said: “We know that people with MS and everyone involved in the trial will be disappointed that a new treatment has not been found. But these results are still incredibly valuable.”

“By conducting the study, we have given the MS community a definitive answer: While simvastatin is a highly effective drug for heart health, it has no additional benefit for people with secondary progressive MS.

“I recommend that people taking simvastatin specifically for MS speak to their doctor if they have any concerns.”

Professor Chataway added: “MS-STAT2 is vital for future MS research and we have demonstrated that we can run large-scale, nationwide trials in progressive MS. We are now focusing our efforts on our multi-arm, multi-stage Octopus trial, which is testing new drugs that have achieved groundbreaking results in other diseases.”

“Ultimately, Octopus will provide people with MS with more treatment options to stop the disease from progressing more quickly.”

MS affects over 150,000 people in the UK and most expect to develop a progressive form of the disease. The disease can be debilitating, exhausting and unpredictable. A previous study – the Phase 2 MS-STAT trial – found that the rate of brain atrophy (shrinkage) was lower in people with secondary progressive MS who took simvastatin compared with those who took a placebo.

Although the results of the Phase 3 MS-STAT2 trial mean that simvastatin is not considered an effective treatment for secondary progressive MS, the study results will contribute to a better understanding of the biology of progressive MS.

The study also highlights the ability of the UK MS community to conduct high-quality, large-scale clinical trials.

Dr Emma Gray, deputy research director at the MS Society, said: “…even though it is not the result we so desperately wanted, it has shown us that we can deliver first-class and well-designed trials that can compete in size and quality with the big pharmaceutical companies.”

“Professor Jeremy Chataway and the study team have made the impossible possible and we are so grateful to the hundreds of MS patients who took part – without them this simply would not have been possible.

“We are on the cusp of a revolution in the treatment of MS. Thirty years ago, there were no treatment options for MS. Today, there are over a dozen treatment options that can help reduce MS attacks. But they do not work for everyone and are not enough to stop MS.

“We will continue to invest in high-quality clinical trials, such as Octopus, which is testing promising drugs against progressive MS.”