Ambroxol, a cough medication, is authorized in Europe for treating respiratory issues and boasts a long history of safety, including its use in high doses and during pregnancy, but it has not been approved in the US or Canada.
Ball and stick model of ambroxol molecules. Image credit: Marina Vladivostok/Chemspider.
Dementia in individuals with Parkinson’s disease can lead to memory loss, confusion, hallucinations, and mood changes.
About half of those diagnosed with Parkinson’s disease will develop dementia within a decade, significantly affecting patients, families, and healthcare systems.
“Our objective was to alter the trajectory of Parkinson’s dementia,” stated Dr. Steven Pasternack, a cognitive neurologist at the Parkwood Institute, St. Joseph’s Healthcare London, and the Robert Institute.
“This preliminary trial offers hope and establishes a robust basis for further research.”
The 12-month clinical trial involved 55 participants suffering from dementia related to Parkinson’s disease.
One group received daily ambroxol, while the other was administered a placebo.
They assessed memory, psychiatric symptoms, and GFAP, markers linked to brain damage in the blood.
The research team noted that ambroxol is safe, well-tolerated, and effectively penetrated the brain.
The placebo group exhibited worsening psychiatric symptoms, while those taking ambroxol remained stable.
Participants with GBA1 genetic mutations showed enhanced cognitive performance with ambroxol treatment.
GFAP levels rose in the placebo group but stayed stable in those using ambroxol, indicating potential brain protection.
“Existing treatments for Parkinson’s disease and dementia focus on symptoms without addressing the root cause,” Dr. Pasternack explained.
“These findings imply that brain function at genetic risk may be preserved, marking a promising new treatment avenue that is currently limited.”
Ambroxol aids an essential enzyme known as Glucocerebrosidase (GCase) linked to the GBA1 gene.
In patients with Parkinson’s disease, GCase levels are frequently reduced. If this enzyme malfunctions, waste builds up in brain cells, resulting in damage.
“This research is crucial as dementia in Parkinson’s disease significantly impacts patients and their families,” remarked Dr. Pasternack.
“If a medication like ambroxol proves beneficial, it can offer true hope and enhance quality of life.”
The results will be published in the journal Jama Neurology.
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Carolina La Silveilla et al. Ambroxol as a treatment for Parkinson’s disease dementia: a randomized clinical trial. Jama Neurology Published online on June 30th, 2025. doi:10.1001/jamaneurol.2025.1687
Source: www.sci.news
