Phase II clinical trial of respiratory drug for treatment of Parkinson’s reveals promising results

Results of a Phase II clinical trial evaluating ambroxol as a potential treatment to slow progression of Parkinson’s (PD) have shown it can effectively cross the blood-brain barrier and increase levels of glucocerebrosidase (GCase) in the brain cells of people with PD. Between January 2017 and April 2018 a Phase II clinical trial was conducted by Professor Anthony Schapira and his research team at University College, London and the Royal Free Hospital. Results were published in The Journal of the American Medical Association (JAMA) Neurology on 13th January, 2020.

Ambroxol is a commonly used medication in Europe, which promotes the clearance of mucus and eases coughing. It is used for the treatment for respiratory diseases, and also has anti-inflammatory properties.

Preclinical experiments suggest that ambroxol may help by bolstering cellular waste disposal systems. In PD, there is evidence to suggest that abnormal proteins are accumulated in cells and are not being disposed of properly. In particular, researchers have found that ambroxol increases levels of a protein called GCase in cells. By increasing levels of GCase, ambroxol allows cells to remove waste more effectively. This would ideally keep cells healthier for longer and therefore slow down the progression of PD.

This study has been funded by CPT in partnership with Van Andel Institute (VAI) and the John Black Charitable Foundation. Further studies will be required to determine if ambroxol is having a disease-modifying impact. CPT, VAI and the John Black Charitable Foundation are now actively exploring the next steps in the clinical testing of ambroxol.