A double‐blind, placebo‐controlled study to assess the mitochondria‐targeted antioxidant MitoQ as a disease‐modifying therapy in Parkinson's disease

TLDR

Mitochondrial oxidative stress is implicated in Parkinson’s disease, and the orally administered antioxidant MitoQ accumulates in mitochondria where it protects against oxidative damage. The study aimed to determine whether two doses of MitoQ would slow Parkinson’s disease progression over 12 months, as assessed by Unified Parkinson Disease Rating Scale scores, in newly diagnosed untreated patients. A double‑blind, placebo‑controlled trial enrolled 128 newly diagnosed untreated PD patients who received two doses of MitoQ or placebo for 12 months, with progression evaluated by clinical rating scales. No difference was observed between MitoQ and placebo on any Parkinson’s disease progression measure, indicating that MitoQ does not slow disease progression.

Abstract

Multiple lines of evidence point to mitochondrial oxidative stress as a potential pathogenic cause for Parkinson's disease (PD). MitoQ is a powerful mitochondrial antioxidant. It is absorbed orally and concentrates within mitochondria where it has been shown to protect against oxidative damage. We enrolled 128 newly diagnosed untreated patients with PD in a double-blind study of two doses of MitoQ compared with placebo to explore the hypothesis that, over 12 months, MitoQ would slow the progression of PD as measured by clinical scores, particularly the Unified Parkinson Disease Rating Scale. We showed no difference between MitoQ and placebo on any measure of PD progression. MitoQ does not slow the progression of PD, and this finding should be taken into account when considering the oxidative stress hypothesis for the pathogenesis of PD.