Generally speaking, I am not a big fan of animal studies... here we see that Ambroxol (found in cough syrups) can alter GCase activity. Important work no doubt, and I guess the rationale was to demonstrate that it crosses the blood brain barrier....
Synapse. 2017 Mar 12. doi: 10.1002/syn.21967. [Epub ahead of print]
Migdalska-Richards A, Ko WK, Li Q, Bezard E, Schapira AH
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 non-human 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.
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