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Oertel, W.H. ; Janzen, A.* ; Henrich, M.T.* ; Geibl, F.F.* ; Sittig, E.* ; Meles, S.K.* ; Carli, G.* ; Leenders, K.L.* ; Booij, J.* ; Surmeier, D.J.* ; Timmermann, L.* ; Strupp, M.*

Acetyl-DL-leucine in two individuals with REM sleep behavior disorder improves symptoms, reverses loss of striatal dopamine-transporter binding and stabilizes pathological metabolic brain pattern-case reports.

Nat. Commun. 15:7619 (2024)
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Isolated REM Sleep Behavior Disorder (iRBD) is considered a prodrome of Parkinson's disease (PD). We investigate whether the potentially disease-modifying compound acetyl-DL-leucine (ADLL; 5 g/d) has an effect on prodromal PD progression in 2 iRBD-patients. Outcome parameters are RBD-severity sum-score (RBD-SS-3), dopamine-transporter single-photon emission computerized tomography (DAT-SPECT) and metabolic "Parkinson-Disease-related-Pattern (PDRP)"-z-score in 18F-fluorodeoxyglucose positron emission tomography (FDG-PET). After 3 weeks ADLL-treatment, the RBD-SS-3 drops markedly in both patients and remains reduced for >18 months of ADLL-treatment. In patient 1 (female), the DAT-SPECT putaminal binding ratio (PBR) decreases in the 5 years pretreatment from normal (1.88) to pathological (1.22) and the patient's FDG-PET-PDRP-z-score rises from 1.72 to 3.28 (pathological). After 22 months of ADLL-treatment, the DAT-SPECT-PBR increases to 1.67 and the FDG-PET-PDRP-z-score stabilizes at 3.18. Similar results are seen in patient 2 (male): his DAT-SPECT-PBR rises from a pretreatment value of 1.42 to 1.72 (close to normal) and the FDG-PET-PDRP-z-score decreases from 1.02 to 0.30 after 18 months of ADLL-treatment. These results support exploration of whether ADLL may have disease-modifying properties in prodromal PD.
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Publication type Article: Journal article
Document type Scientific Article
Corresponding Author
Keywords Parkinsons-disease; Odor Discrimination; Fdg-pet; Identification; Diagnosis
ISSN (print) / ISBN 2041-1723
e-ISSN 2041-1723
Quellenangaben Volume: 15, Issue: 1, Pages: , Article Number: 7619 Supplement: ,
Publisher Nature Publishing Group
Publishing Place London
Non-patent literature Publications
Reviewing status Peer reviewed
Grants Charitable Hertie-Foundation, Frankfurt/Main, Germany
JPB Foundation
Stichting Parkinson Fonds, The Netherlands
Parkinson Fonds, Deutschland
Andre-Leysen-Ulran-Foundation, Belgium