Ambroxol has potential to slow progression of Parkinson’s

Ambroxol is a medication originally designed to clear phlegm and ease coughing in the lungs of patients with respiratory diseases such as bronchitis.

Now, researchers have discovered that this drug has beneficial effects in models of Parkinson’s disease – showing that it may help to remove waste in cells, a function missing in patients with the disease.

Parkinson’s disease is a progressive neurological condition where parts of the brain become progressively damaged over many years by destroying cells that control movement. Typically, people with Parkinson’s have trouble with balance and walking, and they often suffer from involuntary shaking and slow movement. The number of people diagnosed with Parkinson’s in the UK is about 145,000 – around one adult in every 350.

In studies conducted by Professor Anthony Schapira and his research team at UCL Queen Square Institute of Neurology [and funded by MRC, Parkinson’s UK and Cure Parkinson Trust] found that ambroxol increases levels of a protein called glucocerebrosidase in cells.

Glucocerebrosidase, also known as GCase, is located inside small bags of degradative enzymes, called lysosomes, that are found inside cells. Lysosomes are the bags of digestive enzymes that break down rubbish and waste inside a cell.

Mutations of the gene that codes for GCase can result in Parkinson’s, and the activity of this enzyme is also found to be defective in brains of patients with Parkinson’s.

Professor Schapira said the research suggests that by increasing levels of GCase, ambroxol allows cells to remove waste more effectively.

The initial early ‘proof of principle’ study funded by the Cure Parkinson’s Trust involved 17 people with Parkinson’s being treated with ambroxol every day for six months. The results show that ambroxol was well tolerated and that the drug was getting into the brain and increasing the levels of GCase.

“By increasing levels of GCase ambroxol allows cells to remove waste, which would ideally keep cells healthier for longer and could slow down the progression of Parkinson’s,” explained Professor Schapira.

The next stage will be to find the best dose to use for ambroxol and to see if it can slow down Parkinson’s in a large clinical trial. The Cure Parkinson’s Trust and Van Andel Institute are now actively exploring the next step in the clinical testing of ambroxol for Parkinson’s.

Links

• Professor Anthony Schapira 
• UCL Queen Square Institute of Neurology  
• Department of Clinical and Movement Neurosciences 
• Paper in JAMA