Landmark 106 · Trial in people

Simvastatin (PD STAT)

Did not slow Parkinson's in 235 people. Movement scores worsened slightly more on the drug than on placebo.

In short

A UK trial of 235 people found the statin simvastatin, tested because statin use had been linked to lower Parkinson's risk, did not slow progression and, on the main measure, trended slightly worse.

What it is
Trial in people
Outcome
Did not work
When
2022
Size
235 people
Approach
Look beyond the brain
Research lane
Metabolic drug repurposing
Last checked
19 Oct 2026

The research record

This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".

Who

K. N. Stevens, Siobhan Creanor and colleagues (University of Plymouth, per the paper's listed affiliation) across 23 English NHS trusts. Funded jointly by the JP Moulton Charitable Foundation and The Cure Parkinson's Trust.

What happened

Earlier population studies linked statin use to a lower risk of developing Parkinson's, and lab work suggested protection of brain cells. The futility trial ran from March 2016 to May 2020 and enrolled 235 people aged 40-90 with moderate-stage Parkinson's, all with wearing-off (levodopa effect fading between doses), randomised 1:1 to simvastatin or placebo. Dosing was 40 mg a day for a month, then 80 mg for 23 months, with a two-month washout. Of those enrolled, 216 went up to 80 mg and 178 were in the main analysis. The primary measure was the MDS-UPDRS part 3 (motor exam, off medication) at 24 months. Scores worsened 4.5 points on simvastatin and 2.4 on placebo; simvastatin was 1.52 points worse (80% CI −0.77 to 3.80), and the one-sided futility test gave P = 0.006. At 12 months the gap was 2.74 points (95% CI −0.11 to 5.58, P = 0.06), not significant. Myalgia (muscle pain) was the commonest side effect: 36% at 0 mg, 46% at 40 mg and 68% at 80 mg. The investigators called simvastatin futile and said there was no evidence to support a phase 3 trial (JAMA Neurol 2022, DOI below).

Why it moved us toward a cure

It was a clean, well-designed test of a repurposing idea that population data seemed to support. It warns that a link seen in population records does not guarantee that giving the drug will help.

Outcome

Dead end as a general slowing treatment. The investigators said statins might still merit study in selected people, but did not test that.

Sources

How it connects

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Led to

None recorded.

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