Ask the expert: How can clinical trials “slow, stop and reverse” Parkinson’s?

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Author: Saskia MairPublished: 24 September 2020

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Laboratory assistant putting test tubes into the holder

What are clinical trials, why are they so important ­– and what does taking part involve? We talk to Dr Simon Stott and Helen Matthews from The Cure Parkinson’s Trust about how clinical trials help researchers better understand Parkinson’s disease and what potential participants need to know

Why are clinical trials important in Parkinson’s disease research?

Helen Matthews, deputy CEO of The Cure Parkinson’s Trust: Simply, we won’t find a cure without running clinical trials to test new approaches. We can’t find a cure without the thousands of Parkinson’s volunteers who so generously put themselves forward to participate and support research.

Dr Simon Stott, deputy director of research at The Cure Parkinson’s Trust: Any new medical treatment for humans must go through a strict process of clinical evaluation, which investigates safety and efficacy, before health regulators will allow the treatment to be used in the clinic.

We can learn a great deal about the potential biology of Parkinson’s from preclinical models of the condition, but it is not until we actually test our theories in humans that we achieve real insights into the disease. Studies in large clinical cohorts are important in helping to establish a better understanding of the underlying pathology of Parkinson’s and providing better methods of measuring the progression of the condition.

How do trials help scientists and healthcare professionals to understand Parkinson’s disease?

Stott: Clinical trials allow us to build up information about the natural history of the condition – the lived experience – in a manner that can be quantifiably measured. Over time these methods of measuring Parkinson’s are improving and evolving. We have moved beyond simple questionnaires and clinical rating scales towards brain imaging techniques, biological sample, and artificial intelligence approaches. We are collecting more and more data from non-clinical settings using digital and wearable technology, providing a richer definition of Parkinson’s.

Matthews: We are also collecting data on preclinical trials to help us identify and pre-screen potential participants for clinical trials of specific sorts of Parkinson’s. One example of this is PD Frontline, a UK based study, which aims to identify a cohort of people with a genetic form of Parkinson’s.

What sort of breakthroughs have happened as a result of Parkinson’s clinical trials?

Stott: A good example is the cell transplantation studies of the 1980s and 1990s, where individuals were transplanted with dopamine neurons in an effort to replace the cells that had been lost due to Parkinson’s and provide a better quality of life. Some of the participants in those studies have now passed away and their brains have been examined to assess how well the transplanted cell survived. Researchers have found that some of the transplanted healthy cells exhibit evidence of Parkinson’s pathology, which has led to new theories regarding potential cell-to-cell transmission of the disease that could explain the progression of the condition. These theories have resulted in ongoing clinical trials examining therapies targeting this mechanism of transmission.

A recent research report conducted by Parkinson’s research advocates and the research team at The Cure Parkinson’s Trust found that there were 145 registered and ongoing clinical trials for therapeutics targeting Parkinson’s during 2019. And there are many exciting studies within this large collection of trials.

Matthews: Every September, The Cure Parkinson’s Trust and US non-profit Van Andel Institute’s annual International Linked Clinical Trials committee of leading Parkinson’s experts meets to evaluate and prioritise potentially disease modifying drugs that could move into clinical trials. The programme has created an active pipeline of novel and repurposed drugs that we now urgently need to test in clinical trials as it is time to slow, stop and reverse Parkinson’s. We are also so excited to announce a new funding partner, The John Black Charitable Foundation, UK, which will allow us to commit to further potentially curative clinical trials.

Helen Matthews at iLCT

Helen Matthews is deputy CEO of The Cure Parkinson’s Trust.

What does taking part in a trial typically involve?

Stott: Taking part in a clinical trial is a serious matter and it is not a decision that should be rushed or taken lightly. The decision must be based on informed consent, which means being fully aware of the risks of potential negative outcomes of a trial. Before starting any trial, a prospective participant should be given written information on the background of the research, the requirements of the study, and the contact information of all involved parties in the event of an emergency. They should also be made aware that they have the right to stop their involvement in the study at any time without question. Only when a potential participant is comfortable and satisfied with the information or material provided should they be presented with a consent form to fill in.

The nature of the study will determine many of the physical requirements – observational trials might mean quarterly clinical visits, while a clinical trial assessing an experimental therapeutic could require daily self-administration of the drug.

The benefits of taking part in clinical research are many. The opportunity provides the individual with better insights into the nature of their condition. In addition, they are provided with more regular assessment and care, and have the opportunity to ask experts questions about issues that might occur in their daily lives. The risks of participation vary between studies and should be carefully and clearly explained before anyone volunteers to take part.

The teams of investigators conducting the clinical trials are extremely good at what they do, and they are always aware that the safety and wellbeing of the participants comes first.

How has the coronavirus pandemic changed how clinical trials work?

Stott: Before Covid-19, there was an acknowledged need for remote methods of Parkinson’s assessment, allowing for evaluation of symptoms and their progression in real world settings. The Covid-19 situation has served to emphasise that need. Numerous clinical trials have been stalled as a result of the current situation, as participants have been unable to attend assessment sessions.

Matthews: There are lessons we must learn from the Covid-19 clinical trials that were set up and got underway so quickly – applying this agility to Parkinson’s clinical trials could be transformative in terms of the speed, networks, data and participation.

Simon Stott deputy director of research at The Cure Parkinson’s Trust

Dr Simon Stott is deputy director of research at The Cure Parkinson’s Trust.

Need to know

Dr Simon Stott is deputy director of research at The Cure Parkinson’s Trust. He works with the charity’s research team to drive forward preclinical research and funding and on the clinical trials programme that is focused on slowing, stopping and reversing Parkinson’s. He also runs The Science of Parkinson’s, a blog which shares his personal perspective on Parkinson’s research.

Helen Matthews is deputy CEO of The Cure Parkinson’s Trust. She has been involved with the charity since its inception and worked with the late charity co-founder Tom Isaacs. 

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