Overview

• The 12-month randomized Phase II study of 55 Parkinson’s dementia patients found Ambroxol to be safe, well-tolerated and able to reach therapeutic levels in the brain.
• Participants receiving Ambroxol showed stable psychiatric symptoms while those on placebo experienced worsening neuropsychiatric scores.
• High-risk GBA1 gene variant carriers treated with Ambroxol exhibited measurable improvements in cognitive tests.
• GFAP blood levels, a marker of brain cell damage, remained unchanged in the Ambroxol group but rose in placebo participants, indicating potential neuroprotective effects.
• Researchers at Lawson Research Institute plan to begin a larger, cognition-focused clinical trial later in 2025 based on these findings.