香港六合彩中特网

The ASPro-PD trial is a world-first Phase 3 trial aimed at establishing ambroxol鈥檚 potential to slow the progression of Parkinson鈥檚. Following eight years of work with the Parkinson鈥檚 community, driven by UK charity Cure Parkinson鈥檚 in partnership with Van Andel Institute and the John Black Charitable Foundation, this 拢5.5 million trial will investigate the ability of ambroxol to slow the progress of Parkinson鈥檚.

This ground-breaking trial, led by Professor Anthony Schapira at University College London (香港六合彩中特网), will involve 330 people with Parkinson鈥檚 across 10-12 clinical centres in the UK. The trial is placebo controlled and participants will take the drug or placebo for two years. The effectiveness of ambroxol will be measured by its ability to slow the progression of Parkinson鈥檚 using a clinical scale including quality of life and movement. Preparations for recruitment of trial participants have already started.

Ambroxol is one of the drugs prioritised by the International Linked Clinical Trials (iLCT) programme, created and operated by Cure Parkinson鈥檚 and Van Andel Institute. The programme鈥檚 mission is to slow, stop and reverse the progression of Parkinson鈥檚. It aims to significantly reduce the time to bring disease-modifying treatments to clinic for the Parkinson鈥檚 community by testing promising drugs that already have extensive safety data and, in some cases, have been approved by regulators for other medical conditions.

About ambroxol

Ambroxol is a medication that is commonly used in Europe as a treatment for respiratory diseases. It promotes the clearance of mucus, eases coughing and has anti-inflammatory properties.

After reviewing pre-clinical laboratory data from Professor Schapira鈥檚 group at 香港六合彩中特网, a committee of experts brought together by Cure Parkinson's in 2014 prioritised ambroxol for further investigation agreeing that it has the potential to slow or stop the progression of Parkinson鈥檚. This was based on the demonstration that ambroxol could increase the removal of alpha-synuclein, a protein that builds up in Parkinson鈥檚 and is thought to be important in its cause.

Results of the Phase 2 clinical trial, which tested ambroxol in people with Parkinson鈥檚 were published in January 2020 in The Journal of the American Medical Association (JAMA) Neurology. The data showed 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, more effectively.

In addition, the Phase 2 trial showed that ambroxol was safe for people with Parkinson鈥檚 and was well tolerated. This trial was funded and supported by Cure Parkinson鈥檚, Van Andel Institute and the John Black Charitable Foundation.

Funding

This trial will cost 拢5.5 million, which is being funded by Cure Parkinson鈥檚 alongside its strategic partners, Van Andel Institute and the John Black Charitable Foundation (JBCF), and by the Parkinson鈥檚 Virtual Biotech, the drug discovery and development arm of Parkinson's UK. Cure Parkinson鈥檚 is committing to fund 拢2.2 million of the total cost, with the other funders contributing 拢1.1 million each.

Taking Part

Anyone interested in taking part in the ambroxol trial is encouraged to enrol in 鈥楶D Frontline鈥�, a remote study offering online genetic testing for people with Parkinson鈥檚. This can be done now and will help with recruiting people to the ASPro-PD trial, as well as others.聽

Will Cook, CEO of Cure Parkinson鈥檚, said:聽鈥淭his trial is a big step forward in the search to find new treatments for Parkinson鈥檚. 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鈥檚, worldwide. We at Cure Parkinson鈥檚 are working hard - through our efforts within the iLCT programme and in our fundraising efforts 鈥� to increase this number significantly in the next few years, to accelerate our progress towards a cure for Parkinson鈥檚.鈥�

Professor Anthony Schapira, Head of Department of Clinical and Movement Neurosciences at 香港六合彩中特网 Queen Square Institute of Neurology, University College London Hospitals NHS Foundation Trust (香港六合彩中特网H) and the Royal Free London NHS Hospital Trust, and the Chief Principal Investigator of ASPro-PD said:聽 鈥淚 am delighted to be leading this exciting project. This will be the first time a drug specifically applied to a genetic cause of Parkinson鈥檚 disease has reached this level of trial, 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鈥檚, leaders in the field of Parkinson鈥檚, trial design and statistics from the 香港六合彩中特网 CCTU, the MHRA and a consortium of funders led by Cure Parkinson鈥檚, all operating as an effective team to ensure we have reached this stage. We look forward to working with all these groups to ensure successful completion of the study.鈥�

Peter A. Jones, Ph.D., D.Sc. (hon), Chief Scientific Officer at Van Andel Institute said:聽鈥淔or a decade, the International Linked Clinical Trials programme has worked tirelessly to move promising potential therapies for Parkinson鈥檚 into clinical trials. Van Andel Institute is thrilled to continue partnering with Cure Parkinson鈥檚 on this vital initiative and we look forward to a bright future in which slowing or stopping disease progression isn鈥檛 a dream 鈥� it is a reality.鈥�

Darren Moore, Ph.D., Chair of the Department of Neurodegenerative Science at Van Andel Institute and a member of the International Linked Clinical Trials Committee, added:聽鈥淭he movement of ambroxol into a Phase 3 clinical trial is a leap forward in our pursuit of treatments that slow or stop Parkinson鈥檚 progression. Ambroxol has shown promising results in a Phase 2 trial and, because it is a commonly used respiratory medication, it has already gone through rigorous safety testing. Very few potential treatments for Parkinson鈥檚 have聽reached Phase 3 trials, which makes today鈥檚 news that much more exciting. The trial is a testament to the power of collaboration spurred on by the International Linked Clinical Trials initiative.鈥�

Professor David Dexter, Associate Director of Research at Parkinson鈥檚 UK said:聽鈥淲e鈥檙e really pleased that the Virtual Biotech is co-funding this new trial with Cure Parkinson鈥檚 and their strategic partners. People with Parkinson鈥檚 desperately need new and better treatments, and if this trial is a success, ambroxol has the potential to be available in years and not decades. The Parkinson鈥檚 Virtual Biotech is built on a foundation of international collaboration, investing in the most promising drug discovery and development projects. We know that we鈥檒l get results for the global Parkinson鈥檚 community faster by collaborating, not competing. By working together, we can find a cure.鈥�

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Image

• Parkinson's disease. 3D illustration showing neurons containing Lewy bodies small red spheres which are deposits of proteins (alpha-synuclein) accumulated in the brain cells. Credit:聽聽on iStock