Some studies have shown that ambroxol, a cough medicine, may also be effective against Parkinson’s disease. A Phase 3 clinical trial is underway that will try to confirm this.
And drug normally used for cough could be effective in counteracting the Parkinson’s diseaseas suggested by some studies. A new Phase 3 clinical trial at the start it will try to prove it definitively, evaluating the ability of the active ingredient to slow down progression from the neurodegenerative pathology. The drug in question isambroxoloand mucolytic / expectorant con anti-inflammatory properties which has been used since the 70s of the last century, for example to treat bronchitis and promote decongestione of the airways.
That the active ingredient could have far more significant applications than those of a “simple” expectorant was understood only in 2009, thanks to the study “Identification and Characterization of Ambroxol as an Enzyme Enhancement Agent for Gaucher Diseasepublished in the Journal of Biological Chemistry. Scientists led by Professor Don J. Mahuran of the Research Institute – The Hospital for Sick Children of Toronto (Canada) discovered in fact that l’ambroxolo significantly increased the concentrations of a difficult called glucocerebrosidase (Gcase) in patients with Gaucher diseasea lysosomal storage disease of genetic origin that is secondary precisely to glucocerebrosidase deficiency, as explained by Orpha.net.
And what does this have to do with Parkinson’s disease? One of the characteristics of neurodegenerative disease lies in the accumulation of Lewy bodiesor aggregates of one protein call alpha-synuclein. If the levels of this protein increase, those ofdifficult glucocerebrosidase. Starting from this assumption, the scientists of the University of California in Los Angeles led by Professor Anthony Schapira have begun to hypothesize that a high dose of ambroxol could have “kept at bay” the aggregations of alpha-synuclein by increasing those of Gcase, and therefore to counteract the neurodegenerative effects of Parkinson’s disease.
So Professor Schapira and colleagues have started a series of preclinical and clinical studies to verify this hypothesis, observing encouraging signs for the continuation of scientific research. In one Phase 2 study conducted in 2020for example, not only was high doses of ambroxol confirmed to be secure e well tolerated by patients, but also that the active ingredient was able to effectively reach the brain and catalyze the levels of the Gcase protein. The next step will be to understand whether this drug is actually able to effectively fight Parkinson’s disease. So a randomized, placebo-controlled Phase 3 study (called “ASPro-PD”) in which 330 patients will be involved.
The clinical trial will be conducted in the UK with the involvement of a dozen clinical centers and support from the charity Cure Parkinson’s and Van Andel Institute. Using a standardized scale, doctors will determine how well ambroxol will counteract the progression of the disease and improve patients’ symptoms, such as the characteristic tremors. “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 of testing and represents ten years of extensive and detailed work in the laboratory and clinical proof of principle.” Professor Schapira said in a press release. All we have to do is wait for the conclusion of the clinical trial to find out if a cough medicine can effectively combat the devastating neurodegenerative disease, capable of drastically reducing the quality of life of patients and their families.