2024 was another busy year for the research team at Cure Parkinson’s; from the results publication of the phase 2 clinical trial of lixisenatide to the funding of new pre-clinical projects, Cure Parkinson’s has not stopped in its mission to slow, stop or reverse Parkinson’s. Below, we showcase the highlights of 2024 and offer a preview of what’s to come in 2025.
Read on to find updates on the projects below in this article:
- Lixisenatide
- DAPA-PD
- Low-dose lithium
- Exenatide PD-3
- iLCT Pipeline Research Acceleration Grant Programme
- CP-6
- iLCT screening study
- Upcoming trial recruitment for ASPro-PD and EJS ACT-PD
- AZA-PD
- Our next quarterly webinar and our biannual Research Update Meetings
- Research takeaways from 2024 with Dr Simon Stott and No Silver Bullet
2024 updates from Cure Parkinson’s supported clinical trials
Lixisenatide
In September 2023, we got our first insight into the results of the LixiPark phase 2 clinical trial for the GLP-1 receptor agonist lixisenatide. 2024 brought us the full publication of these results, reporting that lixisenatide met its primary endpoint, showing that progression of motor symptoms associated with Parkinson’s slowed in the treatment group when compared to those taking the placebo (dummy drug). These results are encouraging, and Cure Parkinson’s is working closely with the LixiPark investigators to decide the next steps.
DAPA-PD
In February, Cure Parkinson’s and Van Andel Institute (VAI) announced funding for a phase 2 clinical trial of dapansutrile – an anti-inflammatory drug developed by the biotech company Olatec Therapeutics. Researchers believe that by lowering brain inflammation, dapansutrile may be able to slow the progression of Parkinson’s.
Led by Dr Caroline Williams-Gray at the University of Cambridge, this 12 month study will involve 36 people with Parkinson’s and aims to assess if dapansutrile is safe and tolerable. The team hope to use these results to determine whether dapansutrile should be evaluated in a larger phase 3 study.
Low-dose lithium
In May, Cure Parkinson’s and VAI announced funding to support the phase 1b clinical trial of low-dose lithium. Lithium has historically been used to treat bipolar disorder, however it may also hold potential as a disease-modifying therapy for Parkinson’s.
Led by Dr Thomas Guttuso at the University of Buffalo, USA, this trial will recruit 35 people with Parkinson’s (20 of which are supported by the funding from Cure Parkinson’s and VAI) to assess whether lower doses of lithium affect two specific aspects of Parkinson’s biology over 24 weeks. The team at Cure Parkinson’s are excited to see how this trial progresses.
Exenatide-PD3 results
Exenatide-PD3 was a large-scale, two year, phase 3 clinical trial designed to evaluate whether Type 2 diabetes drug exenatide (Bydureon) could slow the progression of Parkinson’s. Building on earlier, smaller studies suggesting potential benefits, this trial used a rigorous double-blind, placebo-controlled design to assess the effects of exenatide on motor symptoms and quality of life.
The trial concluded in early 2024 and unfortunately did not meet its primary endpoint, showing no significant benefit of exenatide compared to placebo. The publication of full results is anticipated in early 2025. We are also waiting on the results from the two Cure Parkinson’s funded sub-studies in this trial, which are expected around this time as well.
2024 highlights from Cure Parkinson’s funded pre-clinical research
iLCT Pipeline Research Acceleration Grant Programme
At the end of 2023, Cure Parkinson’s announced our second round of funding for our iLCT Pipeline Research Acceleration Grant programme. Unlike our open research funding calls, this programme involves Cure Parkinson’s proactively commissioning researchers to test specific drugs in particular laboratory models. The drugs are selected based on recommendations from our International Linked Clinical Trials (iLCT) committee.
In 2024, we announced two new projects as part of this programme. Dr Poonam Thakur from the Indian Institute of Science Education and Research is assessing the neuroprotective effects of probucol and chlorogenic acid, while Dr Sinead O’Sullivan and Professor Donato Di Monte from the German Centre for Neurodegenerative Diseases are investigating the potential of carnosic acid.
CP-6
Dr Marco Fazzari at the University of Pittsburgh, USA, is leading research investigating the therapeutic potential of CP-6, a man-made version of a fatty acid found naturally in the body. The aim of this project is to assess its effects on two Parkinson’s drivers: neuroinflammation and oxidative stress. CP-6 was evaluated by the iLCT committee in 2023, who recommended that further research be conducted before it was considered for a possible clinical trial. We look forward to seeing how this project progresses.
iLCT screening study
For some iLCT-evaluated drugs, more information is needed to aid their progression into clinical testing. To help expedite this process, Professor Heather Mortiboys at the University of Sheffield will be leading work to gather further preclinical data about 100 iLCT-evaluated drugs. They will investigate the impact of these drugs on three drivers of Parkinson’s progression: problems with energy production, waste removal and build-up of harmful proteins. This study is currently in the early phases and we look forward to receiving an update in the spring.
Looking forward to 2025
Upcoming clinical trial recruitment (ASPro-PD and EJS ACT-PD)
In early 2025 we anticipate recruitment will begin for the phase 3 clinical trial of the cough medicine ambroxol (ASPro-PD). The aim of this trial is to evaluate its potential to slow the progression of Parkinson’s. This UK-wide clinical trial will involve two years of treatment with either ambroxol or placebo (dummy drug) in 330 people with Parkinson’s, across 10-12 clinical centres around the UK.
The study is being funded by Cure Parkinson’s in collaboration with its strategic partners, VAI and the John Black Charitable Foundation, and by the Parkinson’s Virtual Biotech, the drug discovery and development arm of Parkinson’s UK.
Alongside this, the Edmund J. Safra Accelerating Clinical Trials for Parkinson’s Disease (EJS ACT-PD) initiative will be starting recruitment in early 2025 as well. This will be a multi-arm, multi-stage (MAMS) clinical trials platform for the continuous testing of potentially disease-modifying treatments for Parkinson’s. This pioneering project will be able to evaluate potential therapies faster than traditional trials by testing multiple drugs at once and comparing them with a single placebo group. This model has previously been successful in advancing therapies in other medical fields, such as oncology.
EJS ACT-PD is being led by Professors Camille Carroll and Tom Foltynie, and will initially involve 1,600 people with Parkinson’s across three different treatment arms of drugs previously prioritised by the iLCT committee.
AZA-PD clinical trial results
Dr Caroline Williams-Gray’s team at the University of Cambridge’s Centre for Parkinson-Plus led a phase 2 clinical trial of azathioprine (AZA-PD), an immunosuppressant medication. The aim of this trial was to assess whether azathioprine was able to slow Parkinson’s progression. The trial concluded in 2024, and the full results are expected to be published in 2025. Cure Parkinson’s is funding a sub-study of the AZA-PD trial to investigate precisely how azathioprine interacts with the immune system in Parkinson’s, with the results to be released alongside the main trial findings.
Quarterly webinars and biannual research update meetings
In 2025, we will continue hosting our regular public research events. Our next Research Update meeting will be held on Monday 24 March at the British Medical Association in London. We will also resume our quarterly webinars in collaboration with the Journal of Parkinson’s Disease and the University of Edinburgh. The next webinar will take place on Wednesday 29 January, with our panellists discussing the recent developments in MAMS initiatives across the world.
Research takeaways from 2024 with Dr Simon Stott and No Silver Bullet
Our Director of Research, Dr Simon Stott was featureded on a webinar on Monday 13 January discussing Parkinson’s research takeaways for 2024 and what to look out for in 2025.
