MDS 2023 | Ambroxol effects in glucocerebrosidase: potential use in Parkinson’s disease

Stephen Pasternak, MD, PhD, FRCPC, Robarts Research Institute, Western University, London, Ontario, Canada, discusses the discovery that ambroxol, which stabilizes glucocerebrosidase, may have potential as a treatment for Parkinson’s disease and other synucleinopathies. While working in a pediatric lysosomal biochemistry lab, Dr Pasternak came across a study showing that a glucocerebrosidase gene mutation was a risk factor for Parkinson’s disease. Knowing that the lab had already found compounds that could stabilize the enzyme, he proposed studying ambroxol in Parkinson’s disease. Though initially met with skepticism, further research confirmed the connection, leading to potential new therapeutic options for patients. This interview took place at the 2023 International Congress of Parkinson’s Disease and Movement Disorders in Copenhagen, Denmark.

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