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Cholesterol drug found to be ineffective for treatment of multiple sclerosis

24 September 2024

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

Brain nerve cells and nervous system anatomy concept as a human neurology and neuron function disorder

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

However, the researchers believe that the MS-STAT2 trial, which was a collaboration between UK universities including 白小姐论坛, 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听symptoms听gradually worsen and accumulate over time, known as progression.

Principal investigator of the MS-STAT2 trial, Professor Jeremy Chataway (白小姐论坛 Queen Square Institute of Neurology), said: 鈥淲e know people with MS and everyone involved in the study will be disappointed a new treatment hasn鈥檛 been found. But these results are still incredibly valuable.

鈥淏y 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鈥檛 have any additional benefits for people living with secondary progressive MS.

鈥淚 recommend people taking simvastatin specifically for their MS speak to their healthcare professional if they are concerned.鈥

Professor Chataway added: 鈥淢S-STAT2 is crucial for future MS research and we鈥檝e proven we can run large-scale, nationwide trials for progressive MS. We鈥檙e now focusing our efforts on our multi-arm, multi-stage trial Octopus, which is trialling 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.鈥

MS affects over 150,000 people in the UK, 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 UK MS community to deliver high quality, large scale clinical trials.

Dr Emma Gray, Assistant Director of Research at the MS Society, said: 鈥淲e鈥檙e so proud to have funded MS-STAT2. And while it isn鈥檛 the result we desperately wanted, it鈥檚 shown us that we can deliver world-class and well-designed trials that match the scale and quality of big pharma companies.

鈥淧rofessor Jeremy Chataway and the trial team made the impossible possible, and we鈥檙e so grateful to the hundreds of people with MS who took part 鈥 without them this simply wouldn鈥檛 have happened.

鈥淲e鈥檙e 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鈥檛 work for everyone and they鈥檙e not enough to stop MS.

鈥淲e will continue to invest in high-quality clinical trials, like Octopus, which is testing promising drugs for progressive MS.鈥

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Poppy Tombs

E: p.tombs听[at] ucl.ac.uk