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Ambroxol Demonstrates Safety and Neuroprotection Signals in Parkinson’s Dementia Trial

Results published in JAMA Neurology show that phase 2 biomarker preservation alongside stable cognitive and psychiatric measures supports a larger, cognition-focused Ambroxol study 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)
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Overview

  • The 12-month, placebo-controlled trial in 55 patients confirmed that daily Ambroxol was safe, well-tolerated and achieved therapeutic concentrations in the brain.
  • Participants on Ambroxol maintained stable neuropsychiatric profiles while those on placebo experienced worsening psychiatric symptoms.
  • Patients carrying high-risk GBA1 gene variants showed measurable cognitive improvements when treated with Ambroxol.
  • GFAP levels, a blood marker of glial cell damage, rose in the placebo group but remained steady in Ambroxol recipients, indicating potential neuroprotective action.
  • A larger, dedicated trial focusing specifically on cognitive outcomes is scheduled for later in 2025 to further evaluate Ambroxol’s disease-modifying potential.