Anthony Schapira, MD, DSc, FRCP, FMedSci, University College London, London, UK delves into the potential of ambroxol for Parkinson’s disease (PD), particularly in patients with mutations in the glucocerebrosidase gene (GBA), a common risk factor for PD. Ambroxol, a common expectorant cough medicine, has been shown to act as a GCase chaperone, increasing GCase activity in brain tissues of rodent and non-human primate models. A small Phase II study, AiM-PD (NCT02941822), revealed that ambroxol is well-tolerated and effectively enters the central nervous system, significantly boosting GCase protein levels in cerebrospinal fluid (CSF) of PD patients. Encouraged by these findings, the ongoing Phase III ASPro-PD study (NCT05778617) aims to assess ambroxol’s potential to slow PD progression over a two-year period in 330 patients, half with a GBA mutation. Interested individuals can contact UCL or enrol through PD Frontline, a UK-wide web-based recruitment platform, to learn more about joining the study and getting their GBA genotype tested. This interview took place at the 18th Annual Congress on Controversies in Neurology (CONy 2024) in London, UK.
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