Professor Anthony Schapira at University College London and the Royal Free Hospital instigated further research. He and his colleagues found that treatment with ambroxol increased the GCase activity in samples of skin cells from people with Parkinson’s.
The Phase 2 Trial of Ambroxol – AIM-PD:
In 2016, Cure Parkinson’s together with Van Andel Institute and the John Black Charitable Foundation, agreed to fund a small phase 2 clinical trial of ambroxol for Parkinson’s (called AIM-PD) led by Professor Schapira. This ran from January 2017 to April 2018. It aimed to lay the foundations for larger trials by answering fundamental questions about the suitability and effectiveness of ambroxol as a potential treatment.
The findings from the trial are by no means definitive because they come from a small ‘proof-of-concept’ study – testing simple biochemical changes in 17 participants. Nevertheless, they add to the evidence that GCase-enhancement is a really promising research theme in our quest to find treatments that slow, stop or reverse the course of Parkinson’s.
Cure Parkinson’s is now working hard with researchers to explore this further and assess robustly the potential of this cough medicine as a future treatment for Parkinson’s. We are also keeping abreast of another clinical trial of ambroxol in Parkinson’s that is underway in Canada as well as several research programmes investigating other ways to target GCase and its actions.
If the evidence in favour of ambroxol and GCase enhancers continues to build, we’ll be at the forefront of driving this into clinical practice for the treatment of patients.