Overview

• A 12-month placebo-controlled trial of once-daily Ambroxol in 55 patients with Parkinson’s disease dementia confirmed its safety and ability to reach targeted brain concentrations.
• Ambroxol recipients maintained stable neuropsychiatric symptoms while those on placebo experienced worsening psychiatric scores.
• Levels of GFAP, a blood marker of brain cell damage, remained stable in the Ambroxol group compared with increases among placebo patients.
• Participants with GBA1 gene variants exhibited preliminary improvements on cognitive assessments when treated with Ambroxol.
• Although Ambroxol is approved in Europe for respiratory use and lacks clearance in North America, funding from the Garfield Weston Foundation supports a larger cognition-focused trial to begin later this year.