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Cough medicine could be used in new treatment for Parkinson's disease

Summary: Ambroxol, a common drug used to treat respiratory illnesses, shows 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 medication currently used to treat respiratory conditions. It promotes the elimination of mucus, relieves coughing and has anti-inflammatory properties.

Preclinical studies, conducted by Professor Schapira at the UCL Queen Square Institute of Neurology, have identified ambroxol as a candidate drug to slow the progression of Parkinson’s.

The results of Professor Schapira’s Phase 2 clinical trial conducted at UCL were published in January 2020 and tested ambroxol in people with Parkinson’s. He found that ambroxol was able to effectively target 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 builds up in Parkinson’s and is thought to be important in its cause), more effectively.

Furthermore, the Phase 2 study showed that ambroxol was safe for people with Parkinson’s and was well tolerated.

The world’s first Phase 3 study, called ASPro-PD, is led by Professor Anthony Schapira and is in partnership with British charity Cure Parkinson’s and the Van Andel Institute – after eight years of working with the Parkinson’s community.

The study will enroll 330 people with Parkinson’s at 10 to 12 clinical centers across the UK. It will be placebo-controlled and participants will take ambroxol for two years.

Ambroxol’s effectiveness will be measured by its ability to slow the progression of Parkinson’s using a scale that includes quality of life and movement. Preparations for recruiting study participants have already begun.

Professor Schapira said: “I am delighted to lead this exciting project. This 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-principles clinical trial.

“The study design is the result of valuable input from people with Parkinson’s, leaders in the field of Parkinson’s, study design and statistics from the UCL Comprehensive Clinical Trials Unit (CCTU), the MHRA and a consortium of funders led by Cure Parkinson’s, all operating as an effective team to ensure we get to that stage.

“We look forward to working with all of these groups to ensure the successful completion of the study.”

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This shows a person holding a pipe.
It shows a brain
He found that ambroxol was able to effectively target the brain and increase levels of a protein known as GCase (glucocerebrosidase). The image is in the public domain

After Phase 2 data from Professor Schapira’s group at UCL found that ambroxol could increase alpha-synuclein clearance, the international Linked Clinical Trials (iLCT) program prioritized research on the drug.

Created and operated by Cure Parkinson’s and the Van Andel Institute, the mission of the iLCT program is to slow, stop and reverse the progression of Parkinson’s. The goal is to significantly shorten the time to bring disease-modifying treatments into 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 Parkinson’s Cure, said: “This study is a major 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 potentially disease-modifying drugs in Parkinson’s worldwide.

“We at Cure Parkinson are working hard – through our efforts in the iLCT program and our fundraising efforts – to increase this number significantly in the coming years to accelerate our progress towards a cure for Parkinson’s.”

About this research news on Parkinson’s disease and neuropharmacology

Author: Poppy Danby
Source: UCL
Contact: Poppy Danby – UCL
Image: The image is in the public domain