UK News

Ambroxol works as a cough medicine by thinning the phlegm build-up caused by many respiratory diseases. A study published in 2009 found that the drug also increased levels of an enzyme called glucocerebrosidase (GCase) in patients who had a rare genetic disorder called Gaucher disease. Patients with this disease are also more likely to develop Parkinson’s, although scientists still aren’t sure why.

A common symptom of Parkinson’s is a build-up of Lewy bodies, groups of a protein called α-synuclein. When the levels of this protein go up, levels of GCase decrease, and the 2009 study proved that ambroxol increased GCase in Gaucher disease patients, so scientists began to consider whether there could be a potential application for the treatment of Parkinson’s.

A smaller human trial has already been undertaken, and results from this appear promising ‒ the drug made it to the brain and increased levels of GCase, possibly decreasing the level of α-synuclein. The drug was also demonstrated to be safe and well-tolerated in Parkinson’s patients, despite the higher than usual dose.

Will Cook, chief executive officer of Parkinson’s charity, Cure Parkinson’s, commented: “Once the ambroxol trial is underway, it will be one of only six phase 3 trials on public record of potentially disease-modifying drugs in Parkinson’s, worldwide. This trial is a big step forward in the search to finds new treatments for Parkinson’s.”

Professor Anthony Schapira, who is leading the study, added: “I am delighted to be leading this exciting project. This will be the first time a drug specifically applied to a genetic cause of Parkinson’s disease has reached this level of trial and represents ten years of extensive and detailed work in the laboratory and in a proof of principle clinical trial.”