Oral ambroxol increases brain glucocerebrosidase activity in a nonhuman primate.
Synapse. 2017-03-17; 71(7): e21967
DOI: 10.1002/syn.21967
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1. Synapse. 2017 Jul;71(7). doi: 10.1002/syn.21967. Epub 2017 Mar 17.
Oral ambroxol increases brain glucocerebrosidase activity in a nonhuman primate.
Migdalska-Richards A(1), Ko WKD(2), Li Q(2)(3), Bezard E(2)(3)(4)(5), Schapira
AHV(1).
Author information:
(1)Department of Clinical Neurosciences, Institute of Neurology, University
College London, NW3 2PF, United Kingdom.
(2)Motac Neuroscience, Manchester, United Kingdom.
(3)Institute of Laboratory Animal Sciences, China Academy of Medical Sciences,
Beijing City, People’s Republic of China.
(4)University de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293,
Bordeaux, F-33000, France.
(5)CNRS, Institut des Maladies Neurodégénératives, UMR 5293, Bordeaux, F-33000,
France.
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.
DOI: 10.1002/syn.21967
PMCID: PMC5485051
PMID: 28295625 [Indexed for MEDLINE]