2018 was a tremendous year for The Cure Parkinson’s Trust (CPT) supported research. In particular, we continued to make considerable progress with our programme of clinical trials:

Clinical trials

CPT's primary goal is to get disease modifying treatments into people with Parkinson’s as quickly as possible. To this end, we are involved with supporting a large number of ongoing clinical trials.

In 2018, news regarding CPT supported clinical trials included:

The completion of the AIM-PD clinical trial of Ambroxol in Parkinson’s. Ambroxol is a commonly used treatment for respiratory conditions, and researchers observed beneficial effects in models of Parkinson’s, which led to this clinical trial being initiated in 2017. The results will be published in 2019.  

The completion of the EPI-589 clinical trial in Parkinson’s. This is a new class of drug that improves energy production and functioning in cells, focusing on mitochondrial dysfunction, and the results will also be available in 2019. Find out more about the trial.

The completion of participant recruitment in the PD-STAT clinical trial of Simvastatin in Parkinson’s. There is evidence that cholesterol reducing statin medication may have beneficial effects in Parkinson’s. In this large, 300+ participant study, we are testing if Simvastatin can help to slow the progression of the condition. 

The continuation of the LIXI-PARK clinical trial in France, which is evaluating the effect of the diabetic drug Lixisenatide in Parkinson's. Lixisenatide is similar to Exenatide - another diabetes drug which has demonstrated positive effects in Parkinson’s. This current study is co-funded by CPT and the Van Andel Institute. 

The continuation of the clinical trial evaluating Liraglutide in Parkinson's. Liraglutide is also a diabetes treatment that is very similar to both Exenatide and Lixisenatide. And again, this study is co-funded by CPT and the Van Andel Institute.  

The continuation of the clinical trial evaluating Deferiprone in Parkinson's. Deferiprone is a drug already used to remove excess iron in the body. Excess iron is thought to play a role in Parkinson's, hence this large clinical trial is evaluating Deferipone in people with Parkinson's. Find out more about the trial 

The initiation of the NILO-PD clinical trial of Nilotinib in Parkinson’s. Recently there has been considerable interest in the effect of the cancer drug Nilotinib and other c-Abl inhibitors for use in Parkinson’s. In 2018, we initiated a large clinical trial in collaboration with the Michael J Fox Foundation and The Van Andel Research Institute. 

Regulatory approval to start a clinical trial of ursodeoxycholic acid (UDCA) for Parkinson’s was given. UDCA is another treatment that boosts energy levels in the brain. Researchers in Sheffield are now initiating a clinical trial of this treatment, with support from the J P Moulton Foundation and CPT. 

In addition to these disease modifying clinical trials, in 2018 CPT also supported other studies, including:

The RAPSODI study - which is a follow-on study of the Ambroxol trial - seeking to provide a platform for targeting neuroprotective medication to those who are developing or at a high risk of developing Parkinson’s disease. Find out more: https://www.rapsodistudy.com/en

The CASPAR is a clinical  trial investigating the efficacy and safety of a treatment (BEN2001) for use against excessive daytime sleepiness in people living with Parkinson’s disease. Find out more https://clinicaltrials.gov/ct2/show/NCT03194217

The Vision in Parkinson’s Study, which aims to used visual tests to better understand differences between people with Parkinson’s. This study is funded by the Wellcome Trust. Find out more here https://medium.com/vision-in-parkinsons).

The Parkinson’s Family Project, which seeks to identify new genes that contribute to Parkinson’s risk and clinical features. Find out more http://www.parkinsonsfamiliesprojects.com/

Preclinical Parkinson’s Research

In addition to ground breaking clinical trials, CPT is also a funder of innovative preclinical research focused on potentially disease modifying therapies that are within 5 years of reaching the clinic.

2018 started with CPT still buzzing from being awarded the inaugural BenevolentAI (https://benevolent.ai/) Award (in collaboration with Parkinson’s UK). This project involves utilising BenevolentAI’s artificial intelligence platform to identify new treatments for Parkinson's. Read more...

The year also started with interesting CPT funded research from scientists at the Van Andel Institute in Michigan, led by Prof. Patrik Brundin. This research explored how the toxic protein alpha synuclein can spread throughout the brain. Read the full paper.

Find out more about Patrk Brundin...

The researchers also wrote an open access review article about their research and how it fits into the wider field of Parkinson’s research. Prof Brundin and colleagues also wrote another review article in which they discuss how targeting cellular energy production in specific ways could be beneficial in the treatment of Parkinson’s.

In May, CPT and 23 other members of the Association of Medical Research Charities (AMRC) announced that they are working in collaboration to develop and launch a joint publication platform. This project will enable researchers to openly and immediately share all of their results from AMRC funded research with the scientific community and beyond. Find out more

In the middle of the year, a CPT co-funded re-analysis of the Phase II Exenatide clinical trial data was published. The results pointed towards certain characteristics in the individuals who responded the best to this diabetes drug that is being re-purposed for Parkinson’s. In 2019, we look forward to the initiation of a large Phase III clinical trial for Parkinson’s. Find out more.

In August, 2018, CPT was delighted to announce that the R S Macdonald Charitable Trust awarded a grant to co-fund the continuation of dopamine replacement stem cell research, being conducted by Dr Tilo Kunath at the Medical Research Centre in Edinburgh. This grant - and previous CPT funding - helped support research conducted by Dr Kunath and his team to produce dopamine neurons that are resistant to the build up of toxic alpha synuclein protein. The results of that research were published in November. Find out more here.

Read Dr Kunath's paper here: https://onlinelibrary.wiley.com/doi/full/10.1111/ejn.14286

In September, CPT and the Van Andel Institute held their annual combination of research meetings. Firstly, the Linked Clinical Trials meeting where an international panel of experts screened a collection of 20+ compounds that have demonstrated potentially disease modifying properties in models of Parkinson’s, prioritising a few for future clinical testing.
Next, the Grand Challenges in Parkinson’s meeting commenced. This meeting was focused on the non-motor features of the condition, with a strong emphasis on the early, pre-motor symptom ‘prodromal’ phase (the period of time before diagnosis). And in parallel with this meeting was the Rallying to the Challenge meeting, which focused on patient involvement in research.

In October, US researchers supported by CPT, published a report suggesting that removal of the appendix can reduce the risk of developing Parkinson’s for individuals who live in rural areas. Read the paper here

In November, Dr Richard Wyse (the Director of Research at CPT) and Prof Patrick Brundin wrote an editorial for an exciting new research report suggesting that inhibition of a cancer-associated pathway (PARP1) has beneficial effects in models of Parkinson’s. Efforts are now being made to take PARP1 inhibitor drugs into the clinic for Parkinson’s. Read this paper here

And finally, in December researchers and advocates in Scotland proposed that individuals with a particular form of Parkinson’s that is associated with a genetic mutation (PARKIN) would be the ideal candidates for investigating the use of cell transplantation for Parkinson’s as these individuals generally do not have the build up of toxic alpha synuclein in their brains. Read the full report. 

2018 was an amazing year for Parkinson's research in general, but particularly for the projects supported by The Cure Parkinson's Trust. We now look forward to a very exciting 2019, in which we will have clinical trial results, new breakthroughs in research, and novel innovations for the Parkinson's community.

Dr Simon Stott - Deputy Director of Research