Ambroxol, the active ingredient in cough syrup, is set to enter a Phase 3 clinical trial early this year in patients with Parkinson’s disease.
The compound has been shown to help clear mucus and displays anti-inflammatory properties. In a Phase 2 trial, it increased levels of glucocerebrosidase (GCase), a protein that helps cells clear waste products—one of which is the protein alpha-synuclein. Ambroxol crossed the blood-brain barrier and was safe and well tolerated in the Phase 2 trial.
Based on these positive results, the international Linked Clinical Trials (iLCT) program has prioritized research of ambroxol. The iLCT is a partnership between Cure Parkinson’s and Van Andel Institute, whose goal is to “commit more therapies to trial to create new, effective treatments that slow, stop, or even reverse disease progression for people living with Parkinson’s.”
The Phase 3 ASPro-PD trial will be led by Dr. Anthony Shapiro of the University College London Queen Square Institute of Neurology. More than 300 patients with Parkinson’s disease will be randomized to treatment with ambroxol or placebo for two years. The drug’s effectiveness will be measured by its ability to slow the progression of Parkinson’s using measures of quality of life and movement.
“This trial is a big step forward in the search to find new treatments for Parkinson’s. Once the ambroxol trial is underway, it will be one of only six phase 3 trials on public record of potentially disease-modifying drugs in Parkinson’s, worldwide,” said Cure Parkinson’s CEO, Will Cook.