The journey to discovering new treatments for Parkinson's (PD) is lengthy and expensive. The Cure Parkinson's Trust (CPT) is determined to fast-track potential new treatments to slow, stop, or reverse PD. Our international Linked Clinical Trials initiative (LCT) 'repurposes' already approved and available drugs and therapies for other conditions, with the potential for treatment of Parkinson's, reducing the time to reach patients by a number of years.

Repurposing Drugs: (A major part of our fundraising is directed towards funding these extremely important trials.)

Annually in September, through our pioneering international Linked Clinical Trials (LCT) initiative, the Linked Clinical Trials committee evaluates multiple drugs currently used, or being developed, in other therapeutic areas, as well as considering natural, non-pharmaceutically based compounds. The committee prioritises drugs and compounds from a dossier of recommendations compiled by Drs. Richard Wyse and Simon Stott (CPT's Director and Deputy Director of Research and Development) and assesses which of these putative treatments would potentially be most suited to move immediately into pilot clinical trials. Aspects considered include known modes of action, safety, blood-brain-barrier penetration, preclinical data in models of PD and relevance to patients along with the possibility to monitor target engagement in the brain (Brundin et al 2013).

LCT is the legacy of Tom Isaacs, founder of The Cure Parkinson’s Trust, and now brings together charities, research organisations and experts from around the world. Since the first annual meeting in 2012, over 2000 drugs have been screened to be repurposed for Parkinson’s. These efforts have been instrumental in bringing directly to trial a number of important drugs with disease modifying potential.

A recent paper (not yet open access) published in the Journal of Parkinson's Disease 'The Linked Clinical Trials initiative (LCT) for Parkinson's disease' by Prof. Patrik Brundin and Dr Richard Wyse, presents the origins of this pioneering initiative:

'...By mid 2019, it is expected that at least 25 drugs prioritised by LCT will have gone to trial, or reached trial completion. It was originally envisaged that several drugs could be tested in parallel, hence “Linked” trials which might share a single control group or similar protocol, to allow for direct comparison. Now, using innovative trial designs this aspect of the programme is also possible.'

Other Recent Papers of Interest

A recent informative paper featuring a number of CPT’s Linked Clinical Trials candidate drugs provides context and an independent rationale for the importance of this programme: 'Drug Repurposing in Parkinson's Disease' ~ Dilan Athauda, Tom Foltynie.

'Can You Teach Old Drugs New Tricks?' This informative article appears in Nature 14 June 2016 by Nicola Nosengo

GLP-1 Agonists

Liraglutide, Lixisenatide, Exenatide and Bydureon belong to a group of safe and well-tolerated drugs called GLP-1 agonists. They are used to treat insulin resistance in type 2 diabetes and in recent years there has been research based evidence to suggest that they show great promise as a new treatment for Parkinson's. A recent meeting conducted by CPT, discussed the further development of trials for these important drugs to ensure that we can find the right routes forward in bringing viable treatments to people with Parkinson’s as quickly as possible.  

Exenatide is a type 2 diabetes drug that  has shown potential neuro-protection in laboratory experiments (*1). Parkinson’s patients given Exenatide for one year continued to show improvements compared to the control group a year after discontinuing treatment. The next phase of these trials, using a weekly dose of the drug Bydureon (AstraZeneca) led by Prof. Tom Foltynie at University College London (UCLH) and funded by the Michael J. Fox Foundation for Parkinson’s Research, has recently finished and a larger phase III trial will begin in the autumn of 2019. Find out more about Exenatide here...

Lixisenatide and Liraglutide are two injectable Diabetes treatments which have been moved into Parkinson’s clinical trials. CPT and Van Andel Research Institute(VARI) are collaborating with the lead investigators in France and the U.S. as well as the pharmaceutical companies Sanofi and NovoNordisk. Two distinct trials have been designed with targeted populations of people with Parkinson’s to investigate this potentially important new approach to treatment.

The Liraglutide trial is recruiting in the US - find out more here


For the last few years the LCT committee has explored whether 'inhibition of c-Abl' would offer a disease-modifying approach for PD. In 2010 a group of researchers published a paper demonstrating that there is increased levels of abl in the Parkinsonian brain, and by blocking the abl protein, c-Abl inhibition could effectively protect models of Parkinson’s disease.

Read further background in the paper here: 'Phosphorylation by the c-Abl protein tyrosine kinase inhibits parkin's ubiquitination and protective function' - Dawson et al. 

C-Abl is a protein that also recently emerged as a therapeutic target showing early promise as a potential disease-modifying therapy when preliminary results were released from a small study at Georgetown University on 'c-Abl inhibition' by Nilotinib in 6 ‘late stage PD patients’ and 6 patients with ‘dementia with Lewy bodies’. While more research must be done to understand its role in PD progression, a lot of activity in the 'c-Abl' field is being carried out to help answer some key questions about its potential.

Based on compelling biochemical evidence and considerable discussion, this expert committee concluded that the use of Nilotinib had significant potential to slow neurodegeneration in PD.” - The Cure Parkinson's Trust Linked Clinical Trials Committee (LCT).

The Cure Parkinson's Trust LCT Committee built a working party comprising the company who own the drug, certain members of the LCT committee and potential funding partners, to design a robust trial to understand the effects of 'c-Abl inhibition' by Nilotinib in people with Parkinson’s.  

Recently, Dr Richard Wyse (CPT), Prof. Patrik Brundin (Van Andel Institute - VARI) and Todd Sherer (Michael J Fox Foundation) - published their article 'Nilotinib – Differentiating the Hope from the Hype' in the Journal of Parkinson's Disease July 2016 discussing the Georgetown safety trial. At the same time, Michael J Fox Foundation, VARI and CPT announced their collaborative partnership to assess the clinical use and development of Nilotinib in Parkinson's. In November 2017 The Michael J Fox Foundation (MJFF) and the Van Andel Research Institute (VARI) announced their recruitment for the Phase lla trial across various centres in the States to test the safety and tolerability of repurposing Nilotinib in Parkinson's disease.

On 25th October 2017 SEONGNAM, a private Korean biotech company, and Neuraly Inc., a startup from Johns Hopkins School of Medicine, announced a collaboration to co-develop novel c-Abl inhibitors for Parkinson's. On 9th March 2017, US biotech Inhibikase announced an MJFF-funded trial to explore the safety profile and brain penetrance of advanced c-Abl inhibitors in pre-clinical models versus other drugs in their class. In addition, Inhibikase is planning another Nilotinib trial, along with another medicine Dasatinib, to investigate its potential in Parkinson's. Also, Georgetown University is moving forward with their further clinical assessments of Nilotinib in PD.

These trials are happening simultaneously across different sites with diverse study designs. The benefit of multiple trials is that researchers will be armed with more data to assess this treatment and help build new pathways faster.  

Further reading:

Uncovering the Promise of c-Abl as a Therapeutic Target: Testing Available Treatments and Supporting New Therapies - Posted by Todd Sherer, PhD, March 09, 2017

UDCA - Ursodeoxycholic acid

This trial has begun and is joint funded by the JP Moulton Foundation and CPT. Find out more about the background to this trial - Professor Oliver Bandmann's presentation at the Parkinson's Academy


EPI-589 is owned by Bioelectron who are running a 'proof of concept' in Parkinson's trial. EPI-589 was designed to treat rare mitochondrial diseases in children. We now know that mitochondrial dysfunction is a hallmark of genetic types of Parkinson’s(*2) and is also thought to be a feature in Parkinson’s where the cause is not known. This trial will help us understand whether this drug can improve mitochondrial function in neurons and, what benefit this has for people with Parkinson’s. 

The next phase of a 2A study has completed and results will be published soon: Phase 2A study of EPI-589 in Parkinson’s.

The study is led by Professor Huw Morris and Dr Vincenzo Libri at the Leonard Wolfson Experimental Neurology Centre at UCLH in London and it is hoped, that this will be the first step in the development of a new Parkinson’s treatment.


Simvastatin already treats high cholesterol but could have neuroprotective qualities in Parkinson’s due to its anti-inflammatory properties. Simvastatin’s effects in Parkinson’s are the subject of a clinical trial that is now underway in 23 centres across the UK led and supported by the University of Plymouth, with joint funding from CPT and the JP Moulton Trust.


Ambroxol was prioritised by the LCT committee in 2014. This anti-mucolytic drug moved into a small proof of concept study at the Royal Free Hospital in London involving a cross section of people with Parkinson’s, including those with genetic forms of the disease who carry a single copy of the glucocerebrosidase enzyme gene mutation (GBA), that is the most common genetic risk factor for PD. It is believed that Ambroxol may help cells to counter the effect of the GBA mutation by increasing levels of glucocerebrosidase in neurons which is understood to enable cells to better dispose of unwanted toxic proteins such as alpha-synuclein. This trial is supported by the John Black Charitable Foundation and VARI.

Find out more about the online Rapsodi study which aims to provide a platform for identifying the early features of Parkinson's and the role of certain genetic mutations in order to develop new, neuroprotective treatments.

Read Professor Schapira's article: 'Glucocerebrosidase Mutations in Parkinson Disease'.


Deferiprone - was prioritised  by the LCT committee in 2012 and a pan-European study is going ahead thanks to funding by the European Commission’s Horizon 2020. Recruitment for the trial is underway. CPT is fully supporting this trial in terms of recruitment and providing information.

The Sky study (Study of Parkinson’s Early Stage - the Jersey Shore Medical Center) is also evaluating Deferiprone in early stage Parkinson's patients and to determine the optimal dose based on motor symptom improvement and safety. SKY is looking to enroll people who were diagnosed with Parkinson’s within the last three years. Existing treatments for Parkinson’s involve giving drugs that increase dopamine in the brain or copy its action.  SKY is taking a different approach by looking at the build-up of iron observed in certain regions of the brain among people with Parkinson’s. It is not known whether this build-up of iron is causing harm to the brain cells, or whether removing it may improve the motor symptoms of the disease or slow its progression. The SKY clinical study aims to address these questions. To find participating centres please visit: FoxTrialFinder (study ID #4412) or (study ID #NCT02728843)

Publications for further information:

*1. Aviles-Olmos I, Dickson J, Kefalopoulou Z, Djamshidian A, Kahan J, Ell P, Whitton P, Wyse R, Isaacs T, Lees A, Limousin P, Foltynie T. 2014. Motor and cognitive advantages persist 12 months after exendatide exposure in Parkinson’s disease. J Parkinson’s Dis 4(3):337–344

*2. Pink1, Parkin, DJ-1 and mitochondrial dysfunction in Parkinson's disease - Mark W Dodson, Ming Guo