Cholesterol drug ineffective for treatment of multiple sclerosis

Results of a world-first phase 3 trial for secondary progressive multiple sclerosis (MS), led by UCL researchers, have shown that the commonly used high-cholesterol drug simvastatin cannot slow disability progression.

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

However, the researchers believe that the MS-STAT2 trial, which was a collaboration between U.K. universities including UCL, the MS Society, the National Institute for Health Research (NIHR), the National MS Society (US) and the NHS, was still a huge milestone in MS research.

MS-STAT2 is the biggest ever trial for secondary progressive MS, involving almost 1,000 participants who currently have limited treatment options.

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

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

“By carrying out the trial, we have provided the MS community with a definitive answer that, while simvastatin is a highly effective drug for heart health, it doesn’t have any additional benefits for people living with secondary progressive MS.

“I recommend people taking simvastatin specifically for their MS speak to their health care professional if they are concerned.”

Professor Chataway added, “MS-STAT2 is crucial for future MS research and we’ve proven we can run large-scale, nationwide trials for progressive MS. We’re now focusing our efforts on our multi-arm, multi-stage trial Octopus, which is trialing emerging medications that have been utterly transformative in other conditions.

“Ultimately, Octopus will lead to more treatments for progression becoming available to people living with MS sooner.”

MS affects over 150,000 people in the U.K., and most expect to develop a progressive form of the condition. It can be debilitating, exhausting and unpredictable. An earlier study—the phase 2 trial MS-STAT—found that the rate of brain atrophy (shrinkage) was reduced in people with secondary progressive MS who took simvastatin compared to those who took the placebo.

While the results of phase 3 trial MS-STAT2 mean simvastatin will not be taken forward as an effective treatment for secondary progressive MS, the trial results will increase understanding of the biology of progressive MS.

The trial also highlights the ability of the U.K. MS community to deliver high quality, large-scale clinical trials.

Dr. Emma Gray, Assistant Director of Research at the MS Society, said, “… while it isn’t the result we desperately wanted, it’s shown us that we can deliver world-class and well-designed trials that match the scale and quality of big pharma companies.

“Professor Jeremy Chataway and the trial team made the impossible possible, and we’re so grateful to the hundreds of people with MS who took part—without them this simply wouldn’t have happened.

“We’re on the cusp of a revolution in how MS is treated. Thirty years ago, there were no treatments for MS. Now there are more than a dozen that can help reduce MS attacks. But these don’t work for everyone and they’re not enough to stop MS.

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