Ambroxol was discovered to be safe and well tolerated throughout a phase 2 clinical investigation into treating Parkinson's.
A a cough suppressant, Ambroxol, thins and decongest mucus. Ambroxol boosts the levels of GCase, a protein that helps cells better eliminate waste proteins like alpha-synuclein, a biomarker for Parkinson’s disease, according to current studies. Ambroxol will start a phase 3 clinical trial in 2023. Researchers began a phase 3 clinical trial to examine its capacity to halt the advancement of Parkinson’s disease.
The World Health Organization estimates that about 8.5 million people worldwide are most likely coping with Parkinson’s in 2019. Parkinson’s disease originates in the substantia nigra, a region of the brain that regulates movement, where nerve cells degenerate or die. Dopamine won’t be produced by these cells when they are damaged or dead. Norepinephrine, commonly known as noradrenaline, may also be damaged in Parkinson’s disease.
Additionally, Lewy bodies are seen in a lot of the brain cells of people with Parkinson’s disease.
Phase 3 clinical study into Ambroxol for treating Parkinson’s:
Between January 11, 2017, and April 25, 2018, 17 Parkinson’s patients who received increasing daily doses of the cough treatment as part of a phase 2 clinical trial of ambroxol were monitored.
The participants’ average age was 60.2 years.
In 2020, JAMA Neurology published the trial’s findings about ambroxol and Parkinson’s.
Each participant in that study underwent a physical, neurological, and ECG evaluation. Researchers obtained the blood samples as well.
Participants received daily dosages of ambroxol ranging from 60 mg to 1.26 g over the course of 186 days.
Over the course of the study, participants received phone check-ins and clinical visits.
At baseline and 186 days, researchers examined the cerebrospinal fluid. At 279 days, a third lumbar puncture was optional.
Throughout the trial, participants reported no negative effects. Moreover, the medication was discovered to penetrate the blood-brain barrier. Researchers also found that participant GCase levels had increased by 35%.
The Van Andel Institute and the U.K. nonprofit organisation Cure Parkinson’s are all collaborators in the Phase 3 trial. It will monitor 330 Parkinson’s patients at 10 to 12 locations throughout the UK.
The most prevalent genetic risk factor for Parkinson’s disease is mutations in the GBA1 gene. Also, Parkinson’s patients without a genetic connection are included in the study.
Ambroxol has to be taken daily for the duration of 2 years, the placebo-controlled trial participants.
Amphroxol and Parkinson’s:
GBA1 mutations are a well-known genetic risk factor for the emergence of Parkinson’s disease. GBA1 contains the blueprints for creating the enzyme glucocerebrosidase (GCase).
According to a 2009 study in the Journal of Blood Chemistry, ambroxol raised GCase levels in Gaucher Disease patients.
GCase function in the brains of Parkinson’s patients will be low with and without GBA1 mutations.
Following it, scientists started to consider ambroxol as a potential treatment for Parkinson’s.
Parvathy Sukumaran 
