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Does taking cholesterol-lowering drugs affect the risk of developing motor neurone disease?

Lay summary by Matthew Chakraverty, reviewed by Dr Naomi Hartopp and an MND lay panel

Background

It is thought that high levels of cholesterol in the blood play a part in the development of motor neuron disease (MND). Research suggests a higher risk of developing the condition if a person has previously had high blood levels of low-density lipoprotein cholesterol (LDL-c). High levels of other substances linked to a fatty diet have also been found to be linked to ALS. Statins reduce cholesterol levels and are already widely used in the prevention of heart disease and strokes. Therefore, it is possible that long term statin use could reduce the risk of people developing MND, but this has not yet been proven. Some existing studies suggest that statins would be helpful, whilst other studies suggest they make no difference and some even suggest they could increase the risk of MND.

Why is the study important?

Unlike previous earlier work, this study gathered data on many patients from Norwegian studies and surveys while considering known risk factors for MND. The authors divided the groups by how long they had been using statins and compared those taking statins with a similar group of people who were prescribed another type of cardiovascular drug that does not affect MND risk. This provided comparable groups of similar people. By accounting for these factors, the results give much more reliable information and presented a clearer picture on the overall benefits of long-term treatments of statins, in terms of MND.

What did the authors do and how did they do it?

Authors used data from several historical health surveys based in Norway. In total they had records of just under half a million patients. The researchers classified those who had been diagnosed with MND, and grouped patients based on how long they had been on statins. They then calculated the risk of each group developing MND by working out the proportion of people who would go on to develop the condition.

What are the results?

Rates of MND initially seemed to be higher in people who had used statins for less than a year. The authors suggest that high cholesterol levels are a symptom of early MND and therefore, these people are likely to have been prescribed statins due to early MND, rather than statins causing MND. In females, there was little change in the chances of developing MND, even with long-term statin use. However, in males taking statins for between 1 and 5 years, the risk of developing MND fell by nearly a quarter and in those that took them for longer than 5 years, this risk fell by over half. Therefore, these results suggest that taking statins has little effect on the risk of developing ALS in females but in males they appear to reduce the risk, and this risk appears to reduce the longer they are on statins.

The exact reasons why males saw greater benefits is unclear. The authors do suggest that as males generally have higher cholesterol levels and are prescribed statins more often, the benefits can be seen more clearly. However, sex specific effects of statins, like a small drop in testosterone levels, cannot be ruled out as possible reasons for the difference.

What do the findings mean going forward for people with the disease?

The study enhances our understanding of the disease, especially in males, and may be helpful in reducing the risk of MND in males. This may prove beneficial to male relatives of people currently with the condition.

It should be kept in mind that statins cannot eliminate the possibility of developing MND as even those who took statins for a prolonged period still developed MND. It also does not mean that females should stop taking statins as they are still useful for preventing other conditions. Further research could now study long term statin use in other populations of MND patients to see if they can prevent MND and in controlled clinical trials to measure their effect on disease progression.

This study can be found at https://www.neurology.org/doi/10.1212/WNL.0000000000214221

Paper title
Duration of Current Statin Use and Amyotrophic Lateral Sclerosis Risk, A Norwegian Population-Based Cohort Study

Lead author
Ola Nakken

Publication details including date of publication
Published 21st October 2025, in Neurology 2025 issue 105 (8) e214221, doi. 10.1212/WNL.0000000000214221