1. Oral ambroxol increases brain glucocerebrosidase activity in a nonhuman primate.
- Author
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Migdalska-Richards A, Ko WKD, Li Q, Bezard E, and Schapira AHV
- Subjects
- Administration, Oral, Animals, Macaca fascicularis, Male, Preliminary Data, beta-Hexosaminidase beta Chain metabolism, Ambroxol administration & dosage, Brain drug effects, Brain enzymology, Central Nervous System Agents administration & dosage, Glucosylceramidase metabolism
- Abstract
Mutations in the glucocerebrosidase 1 (GBA1) gene are related to both Parkinson disease (PD) and Gaucher disease (GD). In both cases, the condition is associated with deficiency of glucocerebrosidase (GCase), the enzyme encoded by GBA1. Ambroxol is a small molecule chaperone that has been shown in mice to cross the blood-brain barrier, increase GCase activity and reduce alpha-synuclein protein levels. In this study, we analyze the effect of ambroxol treatment on GCase activity in healthy nonhuman primates. We show that daily administration of ambroxol results in increased brain GCase activity. Our work further indicates that ambroxol should be investigated as a novel therapy for both PD and neuronopathic GD in humans., (© 2017 The Authors Synapse Published by Wiley Periodicals, Inc.)
- Published
- 2017
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