We would like to share with you a new hope for individuals with Parkinson’s disease.
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Scientists are conducting a Phase III clinical trial to determine if a long-existing cough medication, ambroxol, can slow down the progression of Parkinson’s disease and improve patients’ quality of life. The trial will enroll over 300 people with Parkinson’s disease, some of whom will receive the cough drug ambroxol, and will be placebo-controlled.
Parkinson’s disease is a neurodegenerative condition that causes the brain to become cluttered with the misshapen form of a normally harmless protein called alpha synuclein. This accumulation acts as a driving force in killing or impairing the neurons that provide the brain with dopamine, leading to the movement and muscle control problems seen with Parkinson’s.
Researchers have speculated for several years that ambroxol might be able to help treat Parkinson’s disease and related conditions. The drug has been in use since the late 1970s as a common ingredient in cough medication. It can thin out mucus, allowing people with a cold or other respiratory condition to more easily clear phlegm from their airways and breathe easier. It also reduces inflammation, providing a soothing effect on sore throats.
Studies in the lab and in animals have found that ambroxol can raise levels of glucocerebrosidase (GCase), a protein that helps regulate the brain’s waste clearance system. GCase levels seem to go down as levels of abnormal alpha-synuclein rise in those with Parkinson’s. Rare genetic mutations that cause people to make defective GCase are also known to be a major risk factor for Parkinson’s.
Latest Parkinson’s disease treatment 2022
Small human trials have found that ambroxol can reach the brains of people with moderate Parkinson’s disease and that it can affect the levels of GCase and alpha-synuclein as expected. It also appeared to be safe and well-tolerated, despite needing to be taken in much higher doses than when used as cough medication.
The real test of ambroxol’s effectiveness will begin with the same researchers at University College London conducting a large-scale Phase III trial of the drug. The trial is scheduled to enroll 330 people with Parkinson’s, who will be randomized into either a placebo or treatment group. The volunteers will receive treatment for the next two years, with the researchers monitoring their symptoms and overall quality of life along the way. The team will also check to see whether the drug is more effective in those with GCase mutations, although they are hoping it can still benefit people without the mutations as well.
Even in the best-case scenario, ambroxol is unlikely to represent a true cure for Parkinson’s. However, it could be the first drug known to modify the course of its progression. Current treatments can only help manage people’s symptoms and often become less effective over time as the brain’s production of dopamine deteriorates. The trial is being conducted in partnership with the UK charity Cure Parkinson’s and the Van Andel Institute, a Parkinson’s research center in Michigan.
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