An old cough medicine to slow down Parkinson’s disease? Ambroxol might greatly slow down the progression of the disease.
An important clinical test for Parkinson disease just started at UK. This is to test whether a old cough medicine can slow the progression of this neurodegenerative disease and improve the quality of life of patients. Early studies suggested that this drug may interact with brain proteins important in disease development.
An old cough medicine to slow down Parkinson’s disease?
The molecule in question is ambroxol, it has been used since the end of the 1970s in cough medicines. It thins the mucus, allowing people suffering from cough or other respiratory illness to expectorate better and breathe more easily. It also reduces inflammation, providing a soothing effect to irritated throats. For several years, researchers have believed that ambroxol can help treat the disease of Parkinson and associated diseases.
For Parkinson’s, the brain fills up with a normally harmless protein, alpha-synuclein. Over time, this buildup eventually kills or impairs the neurons that supply the brain with dopamine, leading to the problems with movement and muscle control that are known with Parkinson’s disease. Some research suggested that another protein, glucocerebrosidase or GCase, may play a role in the disease. GCase helps regulate the brain’s waste-scavenging system, and GCase levels appear to decrease as abnormal alpha-synuclein progresses. Rare genetic mutations that can cause people to make deficient GCase can also be a major risk factor for Parkinson’s disease.
Studies in the laboratory and on animals showed that ambroxol can increase GCase levels, which might indirectly decrease levels of abnormal alpha-synuclein in sufferers. Very limited human testing conducted by the University College London showed that ambroxol can reach the brains of mildly ill patients and can affect GCase and alpha-synclein levels as expected. This solution would also be safe and very well tolerated by patients, despite the need to take much higher doses than to treat a cough.
Ambroxol might greatly slow down the progression of the disease
These early studies gave an interesting indication that ambroxol can slow the decline in mobility and other symptoms of patients. But the real test of its effectiveness now begins with this larger-scale Phase III clinical trial, the gold standard of clinical research, if you will. The test should involve 330 patient volunteers, randomly assigned to a treatment group and a placebo. The volunteers will receive the treatment for the next two years and the researchers will closely study their symptoms and quality of life throughout. The team will also seek to find out if this molecule is more effective in patients with GCase mutations, but they hope that it can be beneficial to everyone.
“I am delighted to lead this exciting project. This will be the first time that a drug specifically applied to a genetic cause of Parkinson’s disease has reached this level in clinical testing and it represents ten years of extensive and detailed work in the laboratory and within the rules of clinical testing,” said the team manager, Anthony Schapira, in a statement. The test is run in partnership with the UK charity Cure Parkinson and the VanAndel Institutea Michigan disease research center.
That being said, even in the best of scenarios, ambroxol is very unlikely to be a real treatment for Parkinson’s disease. But it may be the first drug known to halt its progression. Current treatments can only help relieve symptoms and often become less and less effective over time as dopamine production wanes.
