There are serious conditions for which we have no cure, so we keep on searching for anything that might help. Which explains why a cough medicine, yes, you read that right, is being tried out for Parkinson’s.
And not just as symptomatic therapy but as a treatment that could slow down its progression.
Researchers led by a team at University College London will soon launch a phase 3 clinical trial with the active ingredient of cough remedies, ambroxol, to assess its effectiveness in hundreds of people with Parkinson’s.
Ambroxol works by making it easier to cough up phlegm.
Previously, in a 2009 study, ambroxol was found to increase the levels of a brain enzyme called GCase (glucocerebrosidase) in patients with Gaucher disease where there’s a risk of developing Parkinson’s.
In Parkinson’s disease, an abnormal protein (a-synuclein) accumulates in certain regions of the brain. When its levels go up, GCase levels come down.
The 2009 study of Gaucher disease showed ambroxol was able to increase GCase levels, which happen to be low in Parkinson’s. Would ambroxol have the same effect in Parkinson’s and tackle that problematic protein, a-synuclein?
Professor Anthony Schapira and his team at the UCL Institute of Neurology has already performed a small human trial with encouraging results. “I am delighted to be leading this exciting project,” he said.
“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 10 years of extensive and detailed work in the laboratory and in a proof-of-principle clinical trial.”
Ambroxol was able to get into the brain, increasing the levels of GCase, and possibly leading to more
a-synuclein being cleared out – which would be great news for doctors andpatients.
Moreover, ambroxol was found to be safe and well-tolerated.
The next phase of the research is that larger-scale phase 3 multicentre trial in 10-12 clinical centres across the UK, including 330 people who have Parkinson’s disease.
They will be given ambroxol, or a placebo control, for a period of two years, and their symptoms will be monitored to see if the treatment is able to slow the disease’s progression.
Will Cook, CEO of charity Cure Parkinson’s, which is helping to fund the research, said: “Once the ambroxol trial is under way, it will be one of only six phase 3 trials on record of potentially disease-modifying drugs in Parkinson’s worldwide.
“This trial is a big step forward in the search to find new treatments for Parkinson’s.” You can say that again.
