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Oral ambroxol increases brain glucocerebrosidase activity in a nonhuman primate
Author(s) -
MigdalskaRichards Anna,
Ko Wai Kin D.,
Li Qin,
Bezard Erwan,
Schapira Anthony H. V.
Publication year - 2017
Publication title -
synapse
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.809
H-Index - 106
eISSN - 1098-2396
pISSN - 0887-4476
DOI - 10.1002/syn.21967
Subject(s) - glucocerebrosidase , ambroxol , parkinson's disease , enzyme , pharmacology , medicine , disease , chemistry , biochemistry , anesthesia
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.