Cough medicine could be used in new treatment for Parkinson’s disease
summary: Ambroxol, a common drug used to treat respiratory conditions, is showing promise as a treatment for slowing the progression of Parkinson’s disease. Researchers report that ambroxol increases the level of GCase, a protein that allows cells to remove waste proteins including alpha-synuclein.
Ambroxol is a drug currently used to treat respiratory diseases. It promotes the removal of mucus, relieves cough and has anti-inflammatory properties.
Preclinical studies, led by Professor Shapira of the Queen Square Institute of Neurology at UCLA, have identified ambroxol as a candidate drug for slowing the progression of Parkinson’s disease.
The results of a Phase II clinical trial conducted by Professor Shapira and conducted at the University of California in January 2020 were published and tested Ambroxol in people with Parkinson’s disease. It found that ambroxol was able to effectively reach the brain and increase levels of a protein known as GCase (glucocerebrosidase). GCase allows cells to remove waste proteins, including alpha-synuclein (a protein that accumulates in Parkinson’s disease and is thought to be important in its causes), more effectively.
In addition, a phase II trial showed that ambroxol is safe for people with Parkinson’s disease and is well tolerated.
The world’s first Phase 3 trial, called ASPro-PD, is being led by Professor Anthony Shapira and is in partnership with UK charity Cure Parkinson and the Van Andel Institute – after eight years working with the Parkinson’s community.
The trial will include 330 people with Parkinson’s disease at 10-12 clinical centers in the UK. It will be a placebo controlled treatment and participants will take ambroxol for two years.
Ambroxol’s effectiveness will be measured by its ability to slow the progression of Parkinson’s disease using a measure that includes quality of life and movement. Preparations have already begun to recruit participants for the trial.
Professor Shapira said: “I am delighted to be leading this exciting project. This will be the first time that a drug applied specifically to a genetic cause of Parkinson’s disease has reached this level of trial and represents ten years of extensive and detailed work in the laboratory and in a clinical trial proof-of-principle.
“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 Comprehensive Clinical Trials Unit (CCTU), the MHRA, and a group of funders led by Cure Parkinson, all working as an effective team to ensure we get to this point.” .
“We look forward to working with all of these groups to ensure the successful completion of the study.”
After phase II data from Professor Shapira’s group at University College London found that ambroxol could increase alpha-synuclein clearance, the Associated International Clinical Trials (iLCT) program prioritized research into the drug.
Created and operated by the Cure Parkinson Institute and Van Andel, the iLCT program’s mission is to slow, stop, and reverse the progression of Parkinson’s disease. It aims to significantly reduce the time it takes to bring disease-modifying therapies to a clinic for the Parkinson’s community by testing promising drugs that already have comprehensive safety data and, in some cases, have been approved by regulators for other medical conditions.
Will Cook, Cure Parkinson’s CEO, said, “This trial is a huge step forward in the search for new treatments for Parkinson’s disease. Once the Ambroxol trial is conducted, it will be one of only six publicly registered Phase III trials of potentially disease-modifying drugs in the disease.” Parkinson’s, worldwide.
“We at Cure Parkinson are working hard — through our efforts under the iLCT program and in our fundraising efforts — to dramatically increase that number in the next few years, to accelerate our progress toward a cure for Parkinson’s disease.”
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author: Poppy Danby
Contact: Bobby Danby – UCL
picture: The image is in the public domain