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Ambroxol Trial Shows Promise in Slowing Parkinson’s Dementia

Published in JAMA Neurology, the study confirms safety, tolerability, sustained biomarker levels — paving the way for a larger cognition trial later this year.

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St. Joseph’s Health Care on Grosvenor Street in London. Photo taken on Monday June 5, 2023. 
(Mike Hensen/The London Free Press)
Ball-and-stick model of ambroxol molecule. Image credit: Marina Vladivostok / ChemSpider.

Overview

  • The randomized 12-month trial involving 55 Parkinson’s dementia patients demonstrated that daily Ambroxol dosing was safe, well tolerated and achieved target brain concentrations.
  • Psychiatric symptoms and GFAP biomarkers remained stable in the Ambroxol group but worsened in placebo recipients, indicating potential neuroprotective effects.
  • Participants carrying high-risk GBA1 gene variants experienced measurable cognitive gains when treated with Ambroxol.
  • Although Ambroxol has a decades-long safety record as a European respiratory medicine, it is not approved for any use in Canada or the United States.
  • Researchers at Lawson Research Institute are preparing a larger, cognition-focused clinical trial later this year to confirm Ambroxol’s disease-modifying potential.