London, United Kingdom (Jan. 10, 2023) — Following promising results reported at Phase 2 in 2020, a large-scale Phase 3 clinical trial of ambroxol, a drug currently in use to treat respiratory conditions, will start in early 2023.
The ASPro-PD trial is a world-first Phase 3 trial aimed at establishing ambroxol’s potential to slow the progression of Parkinson’s. Following eight years of work with the Parkinson’s community, driven by UK charity Cure Parkinson’s in partnership with Van Andel Institute and the John Black Charitable Foundation, this £5.5 million (approximately $6.6 million USD) trial will investigate the ability of ambroxol to slow the progress of Parkinson’s.
This groundbreaking trial, led by Professor Anthony Schapira at University College London (UCL), will involve 330 people with Parkinson’s across 10–12 clinical centres in the United Kingdom. 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’s 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 prioritized by the International Linked Clinical Trials (iLCT) program, created and operated by Cure Parkinson’s and Van Andel Institute. The program’s mission is to slow, stop and reverse the progression of Parkinson’s. It aims to significantly reduce the time to bring disease-modifying treatments to 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.
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 preclinical laboratory data from Professor Schapira’s group at UCL, a committee of experts brought together by Cure Parkinson’s in 2014 prioritized ambroxol for further investigation agreeing that it has the potential to slow or stop the progression of Parkinson’s. This was based on the demonstration that ambroxol could increase the removal of alpha-synuclein, a protein that builds up in Parkinson’s and is thought to be important in its cause.
Results of the Phase 2 clinical trial, which tested ambroxol in people with Parkinson’s 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’s and was well tolerated. This trial was funded and supported by Cure Parkinson’s, Van Andel Institute and the John Black Charitable Foundation.
This trial will cost £5.5 million (approximately $6.6 million USD), which is being funded by Cure Parkinson’s alongside its strategic partners, Van Andel Institute and the John Black Charitable Foundation (JBCF), and by the Parkinson’s Virtual Biotech, the drug discovery and development arm of Parkinson’s UK. Cure Parkinson’s is committing to fund £2.2 million (approximately $2.6 million USD) of the total cost, with the other funders contributing £1.1 million (approximately $1.3 million USD) each.
Anyone interested in taking part in the ambroxol trial is encouraged to enrol in ‘PD Frontline’, a remote study offering online genetic testing for people with Parkinson’s. This can be done now and will help with recruiting people to the ASPro-PD trial, as well as others. Further information at pdfrontline.com/en.
Will Cook, CEO of Cure Parkinson’s, said:
“This trial is a big step forward in the search to find new treatments for Parkinson’s. 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’s, worldwide. We at Cure Parkinson’s are working hard — through our efforts within the iLCT program and in our fundraising efforts — to increase this number significantly in the next few years, to accelerate our progress towards a cure for Parkinson’s.”
Professor Anthony Schapira, Head of Department of Clinical and Movement Neurosciences at UCL Queen Square Institute of Neurology, University College London Hospitals NHS Foundation Trust (UCLH) and the Royal Free London NHS Hospital Trust, and the Chief Principal Investigator of ASPro-PD said:
“I am delighted to be leading this exciting project. This will be the first time a drug specifically applied 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 proof of principle clinical trial. The study design is the result of valuable input from people with Parkinson’s, leaders in the field of Parkinson’s, trial design and statistics from the UCL CCTU, the MHRA and a consortium of funders led by Cure Parkinson’s, 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.”
Peter A. Jones, Ph.D., D.Sc. (hon), Chief Scientific Officer at Van Andel Institute said:
“For a decade, the International Linked Clinical Trials program has worked tirelessly to move promising potential therapies for Parkinson’s into clinical trials. Van Andel Institute is thrilled to continue partnering with Cure Parkinson’s on this vital initiative and we look forward to a bright future in which slowing or stopping disease progression isn’t a dream — it is a reality.”
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:
“The movement of ambroxol into a Phase 3 clinical trial is a leap forward in our pursuit of treatments that slow or stop Parkinson’s 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’s have reached Phase 3 trials, which makes today’s news that much more exciting. The trial is a testament to the power of collaboration spurred on by the International Linked Clinical Trials initiative.”
Professor David Dexter, Associate Director of Research at Parkinson’s UK said:
“We’re really pleased that the Virtual Biotech is co-funding this new trial with Cure Parkinson’s and their strategic partners. People with Parkinson’s 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’s Virtual Biotech is built on a foundation of international collaboration, investing in the most promising drug discovery and development projects. We know that we’ll get results for the global Parkinson’s community faster by collaborating, not competing. By working together, we can find a cure.”
Video credit: Cure Parkinson’s
About Cure Parkinson’s
Cure Parkinson’s funds and facilitates curative research across the globe. Our funding and innovation through our International Linked Clinical Trials Program has enabled the world’s leading Parkinson’s researchers to collaborate in prioritizing the next generation of drugs for clinical trial. 40% of all drugs that are being researched as possible cures for Parkinson’s have been prioritized by iLCT.
Together we will cure Parkinson’s.
Further information at www.cureparkinsons.org.uk
About Van Andel Institute
Van Andel Institute (VAI) is committed to improving the health and enhancing the lives of current and future generations through cutting-edge biomedical research and innovative educational offerings. Established in Grand Rapids, Michigan, in 1996 by the Van Andel family, VAI is now home to nearly 500 scientists, educators and support staff, who work with a growing number of national and international collaborators to foster discovery. The Institute’s scientists study the origins of cancer, Parkinson’s and other diseases and translate their findings into breakthrough prevention and treatment strategies. Our educators develop inquiry-based approaches for K-12 education to help students and teachers prepare the next generation of problem-solvers, while our Graduate School offers a rigorous, research-intensive Ph.D. program in molecular and cellular biology. Learn more at vai.org.
About University College London (UCL)
UCL is a diverse global community of world-class academics, students, industry links, external partners, and alumni. Our powerful collective of individuals and institutions work together to explore new possibilities.
Since 1826, we have championed independent thought by attracting and nurturing the world’s best minds. Our community of more than 43,800 students from 150 countries and over 14,300 staff pursues academic excellence, breaks boundaries and makes a positive impact on real world problems.
We are consistently ranked among the top 10 universities in the world and are one of only a handful of institutions rated as having the strongest academic reputation and the broadest research impact.
We have a progressive and integrated approach to our teaching and research – championing innovation, creativity and cross-disciplinary working. We teach our students how to think, not what to think, and see them as partners, collaborators and contributors.
For almost 200 years, we are proud to have opened higher education to students from a wide range of backgrounds and to change the way we create and share knowledge.
We were the first in England to welcome women to university education and that courageous attitude and disruptive spirit is still alive today. We are UCL.
About The John Black Charitable Foundation
The John Black Charitable Foundation was set up to support medical research into prostate cancer and Parkinson’s disease in the United Kingdom.
About Parkinson’s Virtual Biotech
A groundbreaking global movement to deliver life-changing new treatments in years not decades.
The Parkinson’s Virtual Biotech uses cutting edge biological and chemical research to come up with new treatments. But it’s driven by people with Parkinson’s, not profit. Collaborative and agile, it adapts successful methods from the business world to deliver new treatments faster. Founded by Parkinson’s UK in 2017, the Parkinson’s Virtual Biotech is now an international programme in partnership with the Parkinson’s Foundation. We believe we’ll get to a cure faster by collaborating, not competing. The next treatment is closer than ever.
To find out more, visit www.parkinsonsvirtualbiotech.co.uk.
Cure Parkinson’s is the operating name of The Cure Parkinson’s Trust. The Cure Parkinson’s Trust is a registered charity in England and Wales (1111816) and Scotland (SCO44368) and is a company limited by guarantee – company number 5539974 (England and Wales).