THE ESSENTIAL
- Nearly 8.5 million people were affected by Parkinson’s disease in 2019, according to the World Health Organization (WHO).
- Parkinson’s disease is characterized by the destruction of dopamine neurons that control movement.
Parkinson’s disease is a degenerative pathology of the brain that results in motor symptoms such as slow movements, tremors, rigidity or imbalance. This condition can also be associated with cognitive, sensory and sleep disorders as well as pain.
Parkinson’s disease is incurable, but drugs, therapies and surgical treatments have been developed to alleviate the disease-related manifestations.
A medicine for respiratory diseases to slow down Parkinson’s
In a preclinical study conducted by the Queen Square Institute of Neurology at University College London (UCL), scientists have observed that ambroxol, a drug recommended to treat respiratory ailments, could slow the progression of Parkinson’s disease.
In 2020, researchers tested ambroxol in patients affected by Parkinson’s disease. In the Phase 2 clinical trial, they noticed that the drug increased levels of GCase (glucocerebrosidase), a protein that allows cells to more efficiently remove protein waste products, especially alpha-synuclein. This protein accumulates in the brain of people affected by the neurodegenerative pathology and causes the appearance of symptoms.
Ambroxol will be evaluated in a new clinical trial
Ambroxol was also well tolerated by patients. The Phase 3 clinical trial, called ASPro-PD, will begin soon and will be led by UCL’s Queen Square Neurological Institute in partnership with UK organization Cure Parkinson’s and the Van Andel Institute. Nearly 330 people affected by Parkinson’s disease will be recruited for this future research. Participants will take ambroxol for two years and their results will be compared to a control group taking placebo.
The efficacy of ambroxol will be assessed by its ability to slow the progression of Parkinson’s disease using a scale including quality of life and movement. “I am delighted to lead this exciting project. This is the first time that a drug applied specifically to a genetic cause of Parkinson’s disease has reached this level of testing. This represents ten years of in-depth and detailed work in the laboratory”said Professor Anthony Schapira, lead author of the study and researcher at UCL’s Queen Square Institute of Neurology.
