A year-long clinical trial has found that Ambroxol, a cough syrup commonly sold across Europe, could potentially slow the progression of dementia in Parkinson's disease patients.
The study, published in JAMA Neurology, monitored 55 participants with Parkinson's dementia and discovered that psychiatric symptoms remained stable in those taking the medication, while the placebo group experienced worsening symptoms.
The research offers hope for the nearly half of Parkinson's patients who develop dementia within a decade of diagnosis.
The condition brings progressively worsening memory loss, confusion, hallucinations and mood changes that affect patients, families and healthcare services alike.
Current therapies for Parkinson's and dementia address symptoms but not the underlying disease
GETTYDuring the trial, researchers divided participants into two groups, with one receiving daily doses of Ambroxol whilst the other received a placebo.
The team assessed participants' memory, psychiatric symptoms and levels of GFAP, a blood marker associated with brain damage.
"Current therapies for Parkinson's disease and dementia address symptoms but do not stop the underlying disease," said neurologist Stephen Pasternak from Western University in Canada.
The small-scale study found that Ambroxol was safe, well-tolerated and successfully reached therapeutic levels in the brain.
Researchers noted that "individuals receiving placebo showed a clinically meaningful worsening in neuropsychiatric symptoms, whereas those receiving Ambroxol remained stable".
The study revealed particularly promising results for patients carrying high-risk genetic variants linked to dementia, who showed improved cognitive performance when taking Ambroxol.
GFAP levels, which indicate brain damage, increased in the placebo group but remained stable in those receiving the medication.
This suggests Ambroxol may protect the brain from accelerated deterioration associated with Parkinson's disease.
Previous research has shown the drug supports glucocerebrosidase (GCase), a key enzyme that is typically low in Parkinson's patients.
When this enzyme fails to function properly, waste accumulates in brain cells, causing damage.
The medication's ability to boost GCase activity could explain its protective effects.
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Ambroxol remains unapproved in the United States and Canada
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Despite its decades-long safety record in Europe, including use at high doses and during pregnancy, Ambroxol remains unapproved in the United States and Canada. The medication is currently prescribed across Europe for respiratory conditions.
"These findings suggest Ambroxol may protect brain function, especially in those genetically at risk. It offers a promising new treatment avenue where few currently exist," Dr Pasternak said.
"If a drug like Ambroxol can help, it could offer real hope and improve lives."
Scientists acknowledged that the drug's clinical effect on cognition was "not confirmed" in this study.
However, they hope the pharmacological data will support larger clinical trials to test Ambroxol's effectiveness in slowing dementia symptoms.
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