2025 Unveiled: Groundbreaking Parkinson’s Cure Research Insights and Future Prospects

2024 was yet another active year for the research team at Cure Parkinson’s; from the publication of phase 2 clinical trial outcomes for lixisenatide to the funding of novel preclinical initiatives, Cure Parkinson’s remains committed to its goal of slowing, halting, or reversing Parkinson’s. Below, we highlight the key moments of 2024 and provide a glimpse into what to expect in 2025.

Continue reading to discover updates on the projects outlined 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 forthcoming quarterly webinar and our biannual Research Update Meetings
• Research highlights from 2024 with Dr. Simon Stott and No Silver Bullet

2024 updates from clinical trials supported by Cure Parkinson’s

Lixisenatide

In September 2023, we received our initial insights into the findings of the LixiPark phase 2 clinical trial for the GLP-1 receptor agonist lixisenatide. The full report of these findings in 2024 indicated that lixisenatide achieved its primary goal, demonstrating that the advancement of motor symptoms related to Parkinson’s was reduced in the treatment group as opposed to those receiving a placebo (inactive drug). These findings are promising, and Cure Parkinson’s is collaborating closely with the LixiPark researchers to determine the following steps.

DAPA-PD

In February, Cure Parkinson’s and Van Andel Institute (VAI) revealed funding for a phase 2 clinical trial of dapansutrile – an anti-inflammatory medication developed by the biotech firm Olatec Therapeutics. Researchers postulate that by diminishing brain inflammation, dapansutrile could potentially decelerate the progression of Parkinson’s.

Directed by Dr. Caroline Williams-Gray at the University of Cambridge, this year-long study will engage 36 individuals with Parkinson’s and seeks to evaluate the safety and tolerability of dapansutrile. The research team anticipates using these findings to establish whether dapansutrile warrants evaluation in a broader phase 3 trial.

Low-dose lithium

In May, Cure Parkinson’s and VAI announced funding to assist the phase 1b clinical trial of low-dose lithium. Lithium has traditionally been utilized for treating bipolar disorder; however, it may also offer potential as a disease-modifying therapy for Parkinson’s.

Led by Dr. Thomas Guttuso at the University of Buffalo, USA, this trial aims to recruit 35 individuals with Parkinson’s (20 of which are backed by funding from Cure Parkinson’s and VAI) to evaluate whether lower doses of lithium influence two particular aspects of Parkinson’s biology over a period of 24 weeks. The team at Cure Parkinson’s is eager to observe the developments in this trial.

Exenatide-PD3 results

Exenatide-PD3 was a large-scale, two-year, phase 3 clinical trial aimed at assessing whether the Type 2 diabetes drug exenatide (Bydureon) could slow the advancement of Parkinson’s. Building on earlier, smaller studies suggesting potential advantages, this trial employed a stringent double-blind, placebo-controlled methodology to evaluate the impact of exenatide on motor symptoms and quality of life.

The trial wrapped up in early 2024 and, regrettably, did not meet its primary objective, exhibiting no significant benefit of exenatide over placebo. The complete publication of the results is expected in early 2025. We are also awaiting the outcomes of the two sub-studies financed by Cure Parkinson’s within this trial, which are anticipated to be released around the same period.

2024 highlights from preclinical research funded by Cure Parkinson’s

iLCT Pipeline Research Acceleration Grant Programme

At the close of 2023, Cure Parkinson’s announced the second round of funding for our iLCT Pipeline Research Acceleration Grant programme. In contrast to our open research funding calls, this programme involves Cure Parkinson’s actively commissioning researchers to assess specific medications in designated laboratory models. The medications are chosen based on the recommendations from our International Linked Clinical Trials (iLCT) committee.

In 2024, we revealed two new initiatives as part of this programme. Dr. Poonam Thakur from the Indian Institute of Science Education and Research is evaluating the neuroprotective properties of probucol and chlorogenic acid, while Dr. Sinead O’Sullivan and Professor Donato Di Monte from the German Centre for Neurodegenerative Diseases are exploring the potential of carnosic acid.

CP-6

Dr. Marco Fazzari at the University of Pittsburgh, USA, is spearheading research into the therapeutic potential of CP-6, a synthetic version of a fatty acid found naturally in the body. The objective of this project is to evaluate its influences on two drivers of Parkinson’s: neuroinflammation and oxidative stress. CP-6 was scrutinized by the iLCT committee in 2023, who recommended further investigation before it could be considered for a potential clinical trial. We are excited to follow the advancement of this project.

iLCT screening study

For some medications evaluated by iLCT, additional information is required to facilitate their progression into clinical examination. To accelerate this process, Professor Heather Mortiboys at the University of Sheffield will lead efforts to accumulate more preclinical data regarding 100 iLCT-evaluated medications. They will assess the effects of these drugs on three factors contributing to the progression of Parkinson’s: issues with energy production, waste elimination, and the accumulation of harmful proteins. This study is currently in its initial phases, and we anticipate receiving an update in the spring.

Looking ahead to 2025

Upcoming clinical trial recruitment (ASPro-PD and EJS ACT-PD)

In early 2025, we expect recruitment to commence for the phase 3 clinical trial of the cough medication ambroxol (ASPro-PD). The goal of this trial is to assess its potential to decelerate Parkinson’s progression. This UK-wide clinical trial will involve two years of treatment with either ambroxol or a placebo (inactive agent) in 330 individuals with Parkinson’s, spanning 10-12 clinical centers across the UK.

This study is backed by Cure Parkinson’s in collaboration with its strategic allies, VAI and the John Black Charitable Foundation, and by the Parkinson’s Virtual Biotech, the drug discovery and development division of Parkinson’s UK.

In tandem with this, the Edmund J. Safra Accelerating Clinical Trials for Parkinson’s Disease (EJS ACT-PD) initiative will also begin recruitment in early 2025. This will establish a multi-arm, multi-stage (MAMS) clinical trials platform for the continuous assessment of potentially disease-modifying treatments for Parkinson’s. This innovative project will enable the evaluation of prospective therapies more swiftly than traditional trials by testing multiple drugs simultaneously and comparing their effects.

them with a solitary placebo cohort. This framework has previously shown promise in promoting treatments in alternative medical domains, such as oncology.

EJS ACT-PD is spearheaded by Professors Camille Carroll and Tom Foltynie and will initially engage 1,600 individuals with Parkinson’s across three distinct therapeutic arms of medications previously prioritized by the iLCT committee.

AZA-PD trial outcomes

Dr. Caroline Williams-Gray’s group at the University of Cambridge’s Centre for Parkinson-Plus directed a phase 2 clinical trial of azathioprine (AZA-PD), an immunosuppressive drug. The purpose of this trial was to evaluate whether azathioprine could decelerate the progression of Parkinson’s. The trial wrapped up in 2024, with complete results anticipated to be published in 2025. Cure Parkinson’s is sponsoring a sub-study of the AZA-PD trial to examine how azathioprine interacts with the immune system in Parkinson’s, with the outcomes scheduled to be disclosed alongside the primary trial results.

Quarterly online seminars and semiannual research summary meetings

In 2025, we will persist in hosting our routine public research gatherings. Our upcoming Research Update meeting is set for Monday, 24 March at the British Medical Association in London. We will also recommence our quarterly online seminars in conjunction with the Journal of Parkinson’s Disease and the University of Edinburgh. The next seminar is scheduled for Wednesday, 29 January, where our panelists will explore recent advancements in MAMS initiatives globally.

Research insights from 2024 with Dr. Simon Stott and No Silver Bullet

Our Research Director, Dr. Simon Stott, will also participate in a webinar on Monday, 13 January to share insights from Parkinson’s research in 2024 and highlight what to anticipate in 2025.

Our advancements towards discovering a cure in 2024 were made possible by your support. To continue fostering our efforts for individuals living with Parkinson’s in 2025 and beyond, please consider making a contribution. Thank you.