Summary: Ambroxol, a drug commonly used to treat respiratory disease, is showing promise as a treatment to slow the progression of Parkinson’s disease. Researchers report that ambroxol increases the level of GCase, a protein that allows cells to remove protein waste, including alpha-synuclein.
Source: UCL
Ambroxol is a drug currently used to treat respiratory conditions. It promotes the elimination of mucus, relieves cough and has anti-inflammatory properties.
Preclinical studies, led by Professor Schapira of the UCL Queen Square Institute of Neurology, identified ambroxol as a candidate drug to slow the progression of Parkinson’s disease.
The results of Professor Schapira’s phase 2 clinical trial carried out at UCL were published in January 2020 and tested ambroxol in people with Parkinson’s disease. He discovered that ambroxol was able to effectively reach the brain and increase levels of a protein known as GCase (glucocerebrosidase). GCase enables cells to more efficiently remove protein waste, including alpha-synuclein (a protein that accumulates in Parkinson’s disease and is thought to be important in its cause).
Additionally, the phase 2 trial showed that ambroxol was safe for people with Parkinson’s disease and was well tolerated.
The first global Phase 3 trial, named ASPro-PD, is led by Professor Anthony Schapira and is in partnership with UK charity Cure Parkinson’s and the Van Andel Institute, following eight years of working with the Parkinson’s community.
The trial will involve 330 people with Parkinson’s disease at 10-12 clinical centers in the UK. It will be placebo-controlled and participants will take ambroxol for two years.
The effectiveness of ambroxol will be measured by its ability to slow the progression of Parkinson’s disease using a scale including quality of life and movement. Preparations for recruiting trial participants have already begun.
Professor Schapira said: “I am delighted to lead this exciting project. It will be the first time that a drug specifically applied to a genetic cause of Parkinson’s disease has reached this level of testing and represents ten years of extensive and detailed work in the laboratory and in a proof-of-principle clinical trial.
“The study design is the result of valuable input from people with Parkinson’s disease, leaders in the field of Parkinson’s disease, trial design and statistics from the UCL Comprehensive Clinical Trials Unit. (CCTU), the MHRA and a consortium of funders led by Cure Parkinson’s, all working as an effective team to ensure we reach this milestone.
“We look forward to working with all of these groups to ensure the study’s success.”
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After phase 2 data from Professor Schapira’s group at UCL revealed that ambroxol can increase the elimination of alpha-synuclein, the international program of linked clinical trials (iLCT) prioritized drug research.
Created and operated by Cure Parkinson and the Van Andel Institute, the iLCT program is on a mission to slow, stop and reverse the progression of Parkinson’s disease. It aims to dramatically reduce the time it takes to bring disease-modifying treatments to the clinic for the Parkinson’s community by testing promising drugs that already have extensive safety data and, in some cases, have been approved by regulators for other medical conditions.
Will Cook, CEO of Cure Parkinson, said: “This trial is a big step forward in the search for new treatments for Parkinson’s disease. Once the ambroxol trial is underway, it will be one of only six publicly registered Phase 3 trials of potential Parkinson’s disease-modifying drugs worldwide.
“At Cure Parkinson’s, we are working hard – through our efforts within the iLCT program and our fundraising efforts – to increase this number significantly over the next few years, to accelerate our progress towards a cure for Parkinson’s disease.
About this Parkinson’s disease and neuropharmacology research news
Author: Danby Poppy
Source: UCL
Contact: Danby Poppy – UCL
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