Cholesterol-lowering statins don’t affect ALS progression: Study

Researchers say ALS shouldn't influence prescription decisions

Written by Marisa Wexler, MS |

A clinician holding a clipboard gestures while speaking to a patient sitting on an examining table.
  • A study showed cholesterol-lowering statins do not significantly affect ALS progression or survival.

  • Statin prescriptions for ALS patients should be based on metabolic needs, not ALS diagnosis.

  • More research is needed, as the study had some limitations.

Statins, medications designed to lower cholesterol, do not meaningfully alter the risk of death or disease progression in people with amyotrophic lateral sclerosis (ALS), according to a study.

The researchers said the findings show that ALS itself should not influence doctors’ decisions about prescribing statins. Instead, statins should be given as needed to ALS patients who need them to treat metabolic issues, they said.

“These findings do not support statin initiation or discontinuation based solely on ALS diagnosis or disease course,” the researchers wrote in the study, “Association between statin use and survival in patients with ALS: A propensity score-matched analysis,” which was published in the Journal of the Neurological Sciences.

ALS is a neurological disease marked by muscle weakness that gets progressively worse over time, ultimately interfering with a person’s ability to perform daily functions. Many people with ALS have high levels of cholesterol and other fatty molecules in the bloodstream, and are prescribed cholesterol-lowering medications such as statins to manage those issues.

Because statins are so widely used, doctors need to understand how they might impact disease progression in people with ALS. Most studies to date have suggested that these medications do not affect ALS survival, but other research has found conflicting results.

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Analysis looks at international database

Scientists in Canada conducted an analysis using data from an international ALS database, the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT), containing data from more than 10,000 ALS patients who took part in clinical trials.

Using a technique called propensity score matching, the researchers identified 131 ALS patients who took statins and matched them with an equal number of patients who were identical in terms of demographics and clinical factors, such as age, disease severity and duration, ethnicity, treatment, and coexisting conditions. The researchers then conducted statistical analyses to compare survival outcomes in the two groups.

Results showed no significant differences in the risk of death after three years between the two groups, indicating that statin use does not meaningfully alter survival outcomes in ALS. The results did not change when the researchers accounted for additional factors that were not included in the propensity matching.

Analyses looking at measures of disease progression likewise suggested no significant effect of statin use.

“Taken together, our findings suggest that statins, irrespective of type, neither worsen nor improve survival or disease progression in ALS,” the researchers wrote. “Statin initiation, continuation, and discontinuation should therefore be guided by cardiovascular and metabolic indications and medication tolerance, and should not be influenced by ALS diagnosis or disease progression, in accordance with current clinical guidelines.”

The scientists noted that their analysis had some limitations. For example, the PRO-ACT database primarily includes ALS patients who have participated in clinical trials, and because trials have strict inclusion criteria, these patients are generally younger and healthier than the overall ALS population.

The analysis also had limited ability to evaluate factors such as statin dosage or treatment timing, which may affect outcomes. That means the question of whether statins affect ALS progression hasn’t yet been fully answered, the researchers said.

“Persistent inconsistencies in literature underscore the need for well-designed prospective studies to more definitively clarify the role of statins and [fat] metabolism in ALS prognosis,” they wrote.

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