A landmark phase 3 clinical trial has begun in the UK. Scientists want to see if it is possible
About ten years ago, researchers studiednew treatments for a condition known as Gaucher disease. This rare genetic disorder results in a deficiency of the enzyme glucocerebrosidase (or GCase). After testing hundreds of already approved drugs, researchers discovered a common ingredient in different cough medications called ambroxol, which increases GCase activity.
Parkinson's disease researchers became interested in this. Low levels of GCase are associated with pathology—this enzyme plays a critical role in helping the brain clear toxic proteins.
Up to 15% of people with Parkinson's diseasediffer in a specific gene mutation that reduces the production of GCase. Also, many patients with Parkinson's disease without a gene mutation still have unusually low levels of GCase. So the scientists decided to test whether ambroxol could work as a treatment for Parkinson's disease if it increases GCase activity.
Preclinical trials were promisingand it soon became clear that the drug could potentially help patients with Parkinson's disease. But two questions remain: whether the drug crosses the blood-brain barrier enough in people to work effectively, and whether patients can tolerate the doses required for the drug to work.
A phase 2 human trial showed thatAmbroxol is tolerated safely in doses high enough to enter the brain and increase GCase levels. Also in 2020, ambroxol was found to reduce levels of the toxic protein alpha-synuclein in patients with Parkinson's disease and potentially improve their motor skills.
The third phase of testing will begin this yearin Great Britain. The study will involve 330 patients with Parkinson's disease, and each participant will be offered either ambroxol or a placebo. The study will last two years.
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