After a year of disruption, clinical research restarted mid-year 2021 and lab-based research got back on track. Cure Parkinson’s is delighted to report that it invested significant funds into research in 2021.
Our recent funding is focused on three key areas:
- Preclinical work ensuring a robust pipeline of relevant drugs to progress into clinical trials
- Infrastructure support to enable the smooth running of those clinical trials
- Further clinical trials of drugs with the potential to slow, stop or reverse Parkinson’s.
New generation diabetes drugs for Parkinson’s
Cure Parkinson’s has long been involved in testing diabetes drugs in people with Parkinson’s because of their neuroprotective effects. Three diabetes drugs currently being clinically tested through our International Linked Clinical Trials programme (iLCT) are exenatide, liraglutide and lixisenatide.
Since the development of exenatide, a new generation of drugs targeting more specific biological pathways in diabetes are available. These ‘second-generation’ drugs are much more effective than exenatide in treating diabetes, and so we think these drugs may also be more effective in slowing down Parkinson’s.
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We are now funding this one year project, led by Dr Dilan Athauda at University College London, which will directly compare these new drugs against exenatide by looking at the protective effects in neurons grown from skin cells taken from people with Parkinson’s. The project will determine which drug has the most neuroprotective effects and at what dose; and this information will then inform researchers of the best drug to be fast-tracked into clinical trials.
Clinical and biomarker data analysis of type 2 diabetes and Parkinson’s
Many studies have indicated that Parkinson’s and diabetes are linked; hence, it is therefore plausible that treatment with diabetes drugs may lessen Parkinson’s severity. This one year data investigation project aims to better understand the links between diabetes and Parkinson’s.
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The East London population has one of the highest levels of diabetes in the UK. People with Parkinson’s taking part in the study will undergo a clinical examination and assessed using a range of measurements. Blood samples will look at differences in the severity of Parkinson’s in people who also have diabetes compared to those without diabetes.
The study team, led by Dr Alistair Noyce at Queen Mary, University of London, will combine information collected from East London with several other large studies. This combination of data will bring together information from hundreds of people to answer questions about the links between diabetes and Parkinson’s with confidence.
Pre-clinical evaluation of Lonafarnib (an iLCT prioritised drug) as a treatment for Parkinson’s
A protein called alpha-synuclein is known to build up and clump together to form abnormal toxic masses known as Lewy Bodies in the brain cells (neurons) of people with Parkinson’s.
A research team at Northwestern University, Michigan under Dr Joseph Mazulli recently discovered a novel method of removing Lewy Bodies and improving neuron health; and Cure Parkinson’s is funding further investigations of this drug.
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The researchers found a biological pathway, called ‘ykt6’, in the neurons that controls the breaking down of the alpha-synuclein protein clumps; and importantly, this pathway could be activated by a drug class called farnesyltransferase inhibitors (or FTI’s). By activating the ykt6 pathway within the nerve cells with the FTI drug, they found this helped breakdown the Lewy Bodies. FTI drugs are a promising approach for Parkinson’s because they easily penetrate the brain and are already being used to treat other diseases, having gone through thorough clinical testing.
The goal of this project is to determine if a clinically safe FTI called Lonafarnib can eliminate abnormal toxic alpha-synuclein in Parkinson’s models. They will establish the optimal dose and use blood biomarkers to track the effectiveness of Lonafarnib to help progress Lonafarnib into clinical trials for people with Parkinson’s.
Funding infrastructure to support clinical trials of the future:
Creation of a Parkinson’s Study Group in the UK
We are delighted to be funding the establishment of a Parkinson’s Study Group in the UK, led by Professor Oliver Bandmann at the University of Sheffield, to enhance the UK’s international reputation for conducting high quality clinical research. This group will strengthen the network of research active clinicians; and synchronise clinical research efforts, streamline contracts, research and development approval processes, support recruitment and provide a strong national support structure for future trials.
PD Frontline: Identifying and grouping 3,000 people with Parkinson’s to support recruitment into upcoming clinical trials
This Cure Parkinson’s funded project began in January 2020. PD Frontline is an online genetic testing and surveying study for people with Parkinson’s and has to date recruited over 1000 participants, creating a clinical cohort of ‘trial-ready’ people for genetic studies to be supported by Cure Parkinson’s in the near future.
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The team, led by Professor Anthony Schapira at University College London, now propose to extend the recruitment and sampling to an additional 2000 people. This will provide DNA identification of gene variants of Parkinson’s in a total of 3000 individuals with Parkinson’s and is estimated to identify approximately 300 people with the GBA gene variant.
Cure Parkinson’s has acquired consent from participants of the study, which means we are now in a position to match people to studies that are better suited for them.
