A drug that has been long used as a medicine for coughs and respiratory illnesses is showing promise in treating Parkinson’s disease. Since it was discovered in 1970s, Ambroxol has remained as an active ingredient in cough syrup mixtures.
In the recent years a new use for the drug has been discovered – treating Parkinson’s.Scientists are investigating the possibility of using Ambroxol to halt the progression of Parkinson’s disease. Raising the hopes of many, the drug has already passed two critical milestone tests in its development.
A multi-institutional team of researchers led by University College London (UCL) reported the results of a small Phase II clinical trial last month which suggested that the ambroxol was safe and well-tolerated in human patients with Parkinson’s disease. The team also has hinted at possible neuroprotective effects that will have to be examined in future studies and subsequent trials.
The results from the research has attracted further funding to continue the evaluation of ambroxol usage in a much larger sample of Parkinson’s patients. The continued research will also study on the links between individual patient genotypes and the disease.
The study on ambroxyl is very crucial as currently we don’t have any treatments to fight the progress of the disease. Simon Scoot, deputy director of research at The Cure Parkinson’s Trust, one of the bodies funding the research program says that “the ambroxol study is important because there are no treatments available for Parkinson’s that slow, stop, or reverse [it]. All of the current medications only deal with the symptoms of the condition – they do nothing to delay the progression of Parkinson’s.”
In the latest open-label trial that lasted over six months, 17 patients with the disease were monitored. The trial was looking into more than just how safe the drug was at the administered levels, it also checked how the drug worked in patients that has a mutated GBA1 (the glucocerebrosidase gene).
GBA1 mutations predisposes people to a greater risk of developing Parkinson’s at a younger age. Scientists believe that this particular mutation inhibits the natural release of glucocerebrosidase proteins (called GCase). GCase performs a clean-up process in the brain which prevents the harmful build-up of a protein called alpha-synuclein. Alpha-synuclein is considered to be as a key reason for the cognitive dysfunction we see in Parkinson’s case.
Previous research has suggested that ambroxol increases the release of GCase proteins and reduces the release of alpha-snuclein. The results need to be replicated in a larger study groups to confirm the findings. But the researchers are very hopeful that things will workout.
Additionally it is suggested that the drug might also improve motor control in Parkinson’s patients. We will have to wait and see how the drug develops in its future testing. Ambroxol’s next stage of evaluation called PD-Frontline has started accepting registrations for patients living in the UK, and this bigger, longer trial will tell us even more about the drug’s viability as a potential treatment.