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. 2024 Mar 27:71:102563.
doi: 10.1016/j.eclinm.2024.102563. eCollection 2024 May.

Safety and efficacy of faecal microbiota transplantation in patients with mild to moderate Parkinson's disease (GUT-PARFECT): a double-blind, placebo-controlled, randomised, phase 2 trial

Arnout Bruggeman  1   2   3   4 , Charysse Vandendriessche  3   4 , Hannelore Hamerlinck  2   5 , Danny De Looze  2   6 , David J Tate  2   6 , Marnik Vuylsteke  3   7 , Lindsey De Commer  8   9 , Lindsay Devolder  8   9 , Jeroen Raes  8   9 , Bruno Verhasselt  2   5 , Debby Laukens  2 , Roosmarijn E Vandenbroucke  3   4 , Patrick Santens  1   2
Affiliations

Safety and efficacy of faecal microbiota transplantation in patients with mild to moderate Parkinson's disease (GUT-PARFECT): a double-blind, placebo-controlled, randomised, phase 2 trial

Arnout Bruggeman et al. EClinicalMedicine. .

Abstract

Background: Dysregulation of the gut microbiome has been implicated in Parkinson's disease (PD). This study aimed to evaluate the clinical effects and safety of a single faecal microbiota transplantation (FMT) in patients with early-stage PD.

Methods: The GUT-PARFECT trial, a single-centre randomised, double-blind, placebo-controlled trial was conducted at Ghent University Hospital between December 01, 2020 and December 12, 2022. Participants (aged 50-65 years, Hoehn and Yahr stage 2) were randomly assigned to receive nasojejunal FMT with either healthy donor stool or their own stool. Computer-generated randomisation was done in a 1:1 ratio through permutated-block scheduling. Treatment allocation was concealed for participants and investigators. The primary outcome measure at 12 months was the change in the Movement Disorders Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) motor score obtained during off-medication evaluations. Intention-to-treat analysis was performed using a mixed model for repeated measures analysis. This completed trial is registered on ClinicalTrials.gov (NCT03808389).

Findings: Between December 2020 and December 2021, FMT procedures were conducted on 46 patients with PD: 22 in the healthy donor group and 24 in the placebo group. Clinical evaluations were performed at baseline, 3, 6, and 12 months post-FMT. Full data analysis was possible for 21 participants in the healthy donor group and 22 in the placebo group. After 12 months, the MDS-UPDRS motor score significantly improved by a mean of 5.8 points (95% CI -11.4 to -0.2) in the healthy donor group and by 2.7 points (-8.3 to 2.9) in the placebo group (p = 0.0235). Adverse events were limited to temporary abdominal discomfort.

Interpretation: Our findings suggested a single FMT induced mild, but long-lasting beneficial effects on motor symptoms in patients with early-stage PD. These findings highlight the potential of modulating the gut microbiome as a therapeutic approach and warrant a further exploration of FMT in larger cohorts of patients with PD in various disease stages.

Funding: Flemish PD patient organizations (VPL and Parkili), Research Foundation Flanders (FWO), Biocodex Microbiota Foundation.

Keywords: Clinical trial; Faecal microbiota transplantation; Gut microbiota; Gut-brain axis; Parkinson's disease.

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Conflict of interest statement

JR has received grants from Beneo, Cargill, Colruyt group, Danone, DSM, J&J, MRM/Prodigest, Nestle, Pfizer, and Takeda; and has received consulting and/or speaking fees from Aphea, Biofortis, DSM, Ferring, GSK, Janssen Pharmaceuticals, Metagenics, MSD, MRM/Prodigest, Nutricia, Sanofi, Takeda, Tsumura. RV has received grants from MRM Health, Prodigest, CellCarta, Evox Therapeutics and Sanofi. All other authors declare no competing interests.

Figures

Fig. 1
Fig. 1
Trial profile.
Fig. 2
Fig. 2
MDS-UPDRS part 3 motor scores (A) and changes in MDS-UPDRS part 3 motor scores (B), by study visit. Data are means for the off-medication state. Error bars represent standard error of the mean. MDS-UPDRS, Movement Disorders Society Unified Parkinson's Disease Rating Scale.
Fig. 3
Fig. 3
Number of radiopaque pellets on day 7 (A) and changes in number of radiopaque pellets (B), by study visit. Higher number of radiopaque pellets correlates to slower colon transit as a marker for constipation (C, D). Data are means. Error bars represent standard error of the mean.
See this image and copyright information in PMC

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