A recently published research report - conducted by Parkinson's research advocates and the research team at The Cure Parkinson's Trust (CPT) – presents an encouraging and useful overview of the current pipeline of agents being clinically tested for the treatment of Parkinson’s.

The investigators analysed a large number of clinical trials (145 studies were included in the analysis) - categorising them into the type of drug being tested and the stage of their clinical development - to highlight interesting trends in how the disease is being targeted and to stimulate discussion and engagement between the research and Parkinson’s patient communities. This report represents one of the first classifications and in-depth analysis of its kind in the Parkinson's clinical trials arena.

As the number of clinical trials focused on Parkinson’s increases, it is important for all of the stakeholders (academic researchers, pharmaceutical industry members, research charities, and people affected by Parkinson’s) to stay abreast of the current developments, to benchmark trends and see emerging promising areas of research. Inclusion of everyone involved is critical as we move forward because it serves to aid in:

• Better therapies being developed to target patient concerns.
• Improved trial designs focused on patient needs.
• Better communication from the research community
• And more informed/engaged patients and families.

“The patient community brings a combination of the lived experience of the disease, a sense of urgency, and an array of career backgrounds and skill sets that can all contribute immensely to the research process. Hopefully, having a clearer view of the trial pipeline will enable greater awareness around opportunities for researcher/patient collaboration” – Sue Buff, Parkinson’s care partner and research advocate.

It is currently very difficult for members of the Parkinson’s community to attain a clear understanding of what is happening within the field of clinical trials for the condition. Large databases, like the US National Institutes of Health’s ClinicalTrials.gov website, provide a search engine to a bewildering array of trials. Websites like the Michael J Fox Foundation’s 'Fox Trial Finder' and Parkinson’s UK 'Take Part Hub' allow individuals to find research studies being conducted within their local area.

However, none provide a concise overview of the broader domain of Parkinson’s research focused on new therapies to treat the condition. It is envisaged that this broad identification of research trends and emerging treatments will facilitate research progression and ultimately it is hoped, a cure for Parkinson's.

“My hope is that this research review will make it easier for those who conduct research, those who fund research, and most importantly, those who volunteer to participate in research, to collaborate more efficiently with the ultimate goal of discovering more effective therapies, and possibly a cure, for the disease." – Gary Rafaloff, Parkinson’s research advocate

Inspired projects by Parkinson’s research advocates like Kevin McFarthing’s 'Parkinson’s Hope List' and Sue Buff’s 'PDTrialTracker' have provided the Parkinson’s community with useful resources that represent excellent sources of information and show examples of proactive advocacy and engagement.

Recently, Kevin and Sue along with another research advocate Gary Rafaloff, have pooled their skills and collaborated with the CPT research team to compile a wide-ranging analysis of the current pipeline of clinical trials evaluating new and emerging therapies for Parkinson’s. Their report has now been published in the Journal of Parkinson’s Disease.

“The real joy of being part of this study was that it was driven by committed and fully engaged research advocates who gave up their free time to provide everyone with an extremely useful resource. It has been inspiring to be part of the project, but full credit belongs to Sue, Kevin and Gary” – Dr. Simon Stott, The Cure Parkinson’s Trust.

Their study involved collecting all of the available information from the ClinicalTrials.gov website regarding ongoing clinical trials for Parkinson’s. After excluding observational and non-drug related studies, they found that there were 145 active registered trials being conducted between January 2019 and January 2020. Of these, 88 trials were focused on symptomatic relief while the remaining 57 trials aimed to slow, stop or reverse the long-term progression of Parkinson’s.

There are 3 phases to the clinical development of drugs: Phase 1 determines the safety of the drug, Phase 2 investigates safety again and looks for signs of efficacy, and Phase 3 seeks to determine the long-term safety and efficacy of the drug being tested. Encouragingly, the team found a wide distribution of studies across these 3 phases, showing a rich and broad range of therapies targeting different approaches to tackling Parkinson’s. From stem cell-based transplantation and boosting the body’s immune system (immunotherapy), to manipulation of the bacteria in the gut and medication which removes excess iron from the brain; many facets of the condition are being tackled.

“The number of clinical trials under way is encouraging for the Parkinson’s community, especially the high proportion of repurposed initiatives that increase the chances of a new therapy becoming available more quickly” – DR. Kevin McFarthing, Parkinson’s research advocate

The researchers noted that 50 of the 145 trials involved the repurposing of drugs that are already being clinically used for other conditions. Repurposing of drugs represents a faster means of getting new treatments through the clinical trial process and into the Parkinson’s clinic. Many of the repurposed trials for Parkinson’s are supported by CPT, such as the exenatide Phase 3 study, the ambroxol Phase 2 study, the UDCA Phase 2 study, and the simvastatin Phase 2 study (the results of which will be announcing later this year).

The results presented in this study highlight the tremendous impact that a modest charity like The Cure Parkinson’s Trust can have on an important global research effort to slow, stop or reverse Parkinson's. Through programmes like the International Linked Clinical Trials initiative, CPT is a driving force in the current drug pipeline for Parkinson’s. As this table demonstrates, fully one third (35%) of disease modifying trials featured in the report involve agents that have been evaluated by the international Linked Clinical Trials Committee, and more than 10% of all the disease modifying trials conducted worldwide are being funded by CPT. As the international Linked Clinical Trials programme enters its 10th year, CPT is committed to increasing that impact by supporting more trials as we seek to cure this condition once and for all.

Tom Isaacs was a massive inspiration for all of us, and his guiding hand still stimulates original initiatives like this important new publication which collates, into a central resource that describes all ongoing clinical trials in Parkinson’s, just as it still energizes us to continue to expand the number of clinical trials we are conducting worldwide via our International Linked Clinical Trials Initiative.

Dr. Richard Wyse, The Cure Parkinson’s Trust

Read the full open access article here. 

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Further reading:

The Journal of Parkinson's Disease - Latest news

The Hope List

PD Trial Tracker

Fox Trial Finder 

Parkinson's UK 'Take Part Hub'.