Parkinson’s UK to co-fund a phase 3 stage clinical trial with Cure Parkinson’s

Through the Parkinson’s Virtual Biotech, we’re investing £1.1m to co-fund a study looking at whether a cough medicine, ambroxol, has the potential to slow the progression of Parkinson’s.

Whilst past trials have shown promise, there is still more research needed to show if ambroxol can be a safe and effective treatment for Parkinson's. This includes safe testing of dosages and frequencies. Parkinson's UK does not recommend you take any medications without a prescription. If you have any questions, please contact our research mailbox at [email protected] 

The Parkinson’s Virtual Biotech is a groundbreaking global movement to deliver life-changing new treatments in years, not decades. And we believe we can only do this through collaboration.

That’s why we’re joining forces with Cure Parkinson’s, alongside its strategic partners Van Andel Institute (VAI) and the John Black Charitable Foundation (JBCF) to co-fund a clinical trial looking at the potential of a cough medicine called ambroxol to treat Parkinson’s.

The ASPro-PD trial is a world-first phase 3 trial of ambroxol. Driven by Cure Parkinson’s, following 8 years of work with the Parkinson’s community, this £5.5m trial offers hope that a drug to slow the progression of Parkinson’s may be on the horizon.

What is ambroxol?

In 2009 researchers discovered that a medicine commonly used to treat coughs and sore throats, ambroxol, could also be used to treat Parkinson’s.

Ambroxol boosts levels of an enzyme called GCase, which is known to help clear away waste products which have gathered in brain cells. In Parkinson’s, a build up of a troublesome protein called alpha-synuclein is often seen in the brain tissue. It’s thought that ambroxol may help improve the body’s ability to clear away these clumps of alpha-synuclein and prevent damage to brain cells.

Results from a trial of the drug in 2020 funded and supported by Cure Parkinson’s and its strategic partners, showed that ambroxol was safe for people with Parkinson’s to take. It also showed that the drug could reach the area of the brain needed to boost GCase.

The new trial will test the drug in a larger group of people with Parkinson’s. It will compare it to a dummy drug to see if it can slow the progression of the condition. Cure Parkinson’s has been the driving force behind progress towards this trial through its International Linked Clinical Trials Programme (iLCT) alongside VAI. 

Read more about the International Linked Clinical Trials Programme.

Next steps

There’s some evidence to show that people that have a change in one gene associated with Parkinson’s might benefit from treatment with ambroxol. The team are inviting anyone who would be interested in joining the ambroxol trial to register now for the PD Frontline study and use their online genetic testing service.

Find out more about PD Frontline on our Take Part Hub.

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 its strategic partners. People with Parkinson’s desperately need new and better treatments.

"This is the first large phase 3 trial that we have funded, and if this 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."

Dr Arthur Roach, Director of Research at Parkinson's UK, said:
"This is an exciting phase for the Parkinson’s Virtual Biotech. We’ve been growing steadily and are starting to see the first successes from the early projects we’ve invested in. But we’ve always known that there is much more to be done than we can manage on our own.

"Thanks to collaborations like this one with Cure Parkinson’s and partners, and through our international partnership with the Parkinson’s Foundation, we’re making this happen. Working together, we can deliver much needed new treatments, driven by the needs of people with Parkinson’s."

Update December 2023: Delays to the ASPro-PD study

The ASPro-PD study is not yet ready to recruit participants. This is due to delays in producing the correct formulation of the ambroxol drug for the study. This process has been under way for a while but has taken longer than anticipated. The reason the drug is being redesigned from the previous study is to prevent participants having to take an excessive amount of pills a day. At this time we don’t have an exact timeline to share, but we are in close communication with Cure Parkinson's and other partners to ensure we are providing timely updates. We hope to share the next update in the new year.

The Parkinson's Virtual Biotech

The Parkinson’s Virtual Biotech is a groundbreaking global movement to deliver life-changing new treatments in years not decades.