overview: Ambroxol, a common drug used to treat respiratory disorders, shows promise as a treatment for slowing the progression of Parkinson’s disease. increase. GCase is a protein that enables cells to remove waste proteins, including α-synuclein.
Ambroxol is a drug currently used to treat respiratory diseases. It promotes the elimination of mucus, relieves coughs and has anti-inflammatory properties.
A preclinical study led by Prof. Schapira at UCL Queen Square Neurology Institute identified ambroxol as a candidate drug to slow the progression of Parkinson’s disease.
Results from a Phase 2 clinical trial conducted by Professor Schapira at UCL were announced in January 2020, testing ambroxol in patients with Parkinson’s disease. It turns out that ambroxol can effectively reach the brain and increase levels of a protein known as GCase (glucocerebrosidase). GCase allows cells to more effectively remove waste proteins such as alpha-synuclein, a protein that accumulates in Parkinson’s disease and is believed to be causatively important.
Additionally, a Phase 2 study showed that ambroxol is safe and well-tolerated in patients with Parkinson’s disease.
The world’s first Phase 3 trial, named ASPro-PD, is led by Professor Anthony Schapira in partnership with UK charities Cure Parkinson’s and the Van Andel Institute.
The trial will enroll 330 people with Parkinson’s disease at 10-12 clinical centers in the UK. A placebo-controlled participant will take ambroxol for her 2 years.
Ambroxol’s efficacy is measured by its ability to slow the progression of Parkinson’s disease using measures that include quality of life and movement. Preparations for recruitment of clinical trial participants have already begun.
Professor Schapira said: This is the first time that a drug specifically applied to the genetic cause of Parkinson’s disease has reached this level of testing, and is based on his decade of extensive and detailed research in the laboratory and proof-of-principle clinical trials. represents.
“This study design benefited from valuable input from trial design and statistics from a consortium of funders led by Parkinson’s patients, leaders in the field of Parkinson’s disease, the UCL Comprehensive Clinical Trials Unit (CCTU), MHRA, Parkinson’s Disease Cure. We are working as an effective team to make sure we reach this stage.
“We look forward to working with all these groups to ensure the success of our research.”
The international Linked Clinical Trials (iLCT) program prioritized research into the drug after phase 2 data from UCL’s Professor Schapira’s group found that ambroxol could enhance the elimination of alpha-synuclein.
Created and operated by Cure Parkinson’s and the Van Andel Institute, the iLCT program’s mission is to slow, stop and reverse the progression of Parkinson’s disease. Time to bringing disease-modifying therapies to the clinic for people with Parkinson’s disease by testing promising agents that already have extensive safety data and, in some cases, regulatory approval for other conditions It is intended to be significantly shortened.
Cure Parkinson’s CEO Will Cook said: Once trials of ambroxol begin, this marks him one of six phase III trials of his publicly documented worldwide for a potential disease-modifying drug for Parkinson’s disease. Become.
“Cure Parkinson’s is committed to significantly increasing this number over the next few years and accelerating progress towards the treatment of Parkinson’s disease through our efforts within the iLCT program and our fundraising efforts.”
About this Parkinson’s disease and neuropharmacology research news
author: poppy danby
contact: Poppy Danby – UCL
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