Publication date: Jan 14, 2020
In a small, open-label, single-center study of Parkinson’s patients, ambroxol crossed the blood-brain barrier and boosted β-glucocerebrosidase (GCase) enzyme protein levels, reported Anthony Schapira, MD, DSc, of University College London, and colleagues in JAMA Neurology.
“The results of the AiM-PD study indicate that ambroxol is safe, well tolerated, and able to increase GCase levels in the spinal fluid of people with Parkinson’s,” Schapira told MedPage Today.
Although GCase activity is reduced in Parkinson’s patients with and without GBA1, it’s lower in people with the mutations.
In this nonrandomized, non-controlled trial, Schapira and colleagues treated 18 Parkinson’s patients with escalating doses of oral ambroxol to 1. 26 g per day (420 mg 3 times a day) from January 2017 to April 2018.