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AD/PD 2023 | The patient experience in cell- and gene-therapy trials for Parkinson’s disease

Advanced therapy medicinal products (ATMPs) are of great interest for the treatment of Parkinson’s disease (PD). Numerous cell- and gene-based therapies are currently undergoing clinical trials in this setting. Emma Lane, PhD, Cardiff University, Cardiff, UK, explains the work being done to understand both the participant’s and their support network’s experience within these clinical trials. The LEARN study conducted retrospective interviews with participants from the intraputamenal GDNF and the TRANSEURO dopamine cell replacement trials and their family members/care partners. The results showed a majority of positive experiences with participants very aware of the trial processes. The study also found that, with long trials, it was important to make participants aware of the processes they may go through at the end of the trial. This was because the end of the trial experience was flagged as a difficult one due to the loss of close interaction with healthcare professionals. Another point from the trials was that some patients and their support networks found brain imaging assessments and the assessment of motor function without medication difficult. This study was important as the collective experience is critical to better designing later trials and providing better information for the patient to ensure good study retention. This interview took place at the AD/PD™ 2023 congress in Gothenburg, Sweden.

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Transcript (edited for clarity)

Advanced therapeutic medicinal products are cell- and gene-therapies which are being planned as interventions for the treatment of Parkinson’s disease. These are quite invasive interventions and cover a whole range of different types of approaches. But what we found was that there’s very little known about what participants in those trials may feel about what they experience during quite complex trials and also what their journey is like and also what the journey of their partners or support network around them is like as they go through these trials...

Advanced therapeutic medicinal products are cell- and gene-therapies which are being planned as interventions for the treatment of Parkinson’s disease. These are quite invasive interventions and cover a whole range of different types of approaches. But what we found was that there’s very little known about what participants in those trials may feel about what they experience during quite complex trials and also what their journey is like and also what the journey of their partners or support network around them is like as they go through these trials. So the work that we’ve done is to set out to try and understand in these invasive complex trials in Parkinson’s disease what that experience actually is for those living it.

So the work that we’ve been doing has largely been retrospectively interviewing the participants on the GDNF study, which took place in Bristol in the United Kingdom and involved intracranial implantation of cannulae infusing GDNF directly into the brain. And we’ve also interviewed participants on the TRANSEURO study, which was a clinical trial of fetal cell transplantation led by Roger Barker at Cambridge University. So we’ve interviewed both cohorts of participants in those trials and we’ve also opened up the opportunity to interview to family members and those who have supported those participants within the trials, so we can try and get a good picture of what the lived experience might be like. Importantly, what we’re trying to capture is the range of experience of people on the trials. It’s very easy for individual experiences to be captured, for patient advocates to report their experiences and we often do this. We often bring individual people into symposia or to conferences to talk about their individual lived experience. What we don’t do is look at the breadth of experience across all the participants to try and capture all those nuances to determine how common that one experience was. Who had a bad experience going into an MRI or PET scanner, who had a positive experience? Was it universally a poor experience and is that consistent across trials or is this site specific? Is this based on individual experiences? So it’s trying to look more at the collective experience rather than honing in on single individuals so that we can try and use that information to better design trials in the future and avoid some of those points or provide better information to better inform participants as they go through the trial and therefore hopefully ensure good retention and good experience on trials.

So the majority of participants had really positive experiences on the trials, which is really important and they were very aware of the processes they consented to. But what we found was on very long trials, there might be a need to remind participants of their role as participants, not patients, within the trial and to make them aware of the process that they may go through at the end of the trial. The end of trial experience can be quite challenging for some participants because if they’ve had a lot of communication with healthcare professionals, that’s very attractive to them. They receive a lot of input in terms of managing their Parkinson’s. And then if you transition into being a patient, that close interaction that you’ve had with healthcare professionals disappears and you go back to far fewer appointments, far fewer interactions with healthcare professionals. And that can be somewhat disconcerting or challenging for participants. So there’s a real transition period at the end of a trial. And there are also specific points during trials that some participants find challenging. That includes assessments of motor function off their medication and also some of the brain imaging were very difficult experiences. Some participants have no problem at all going through the brain imaging, whether that’s on or off medication. But for some patients that was a really challenging experience and also for their partners or support partners who were with them seeing their loved ones fully off their medication, in some cases for the first time, was emotionally quite a difficult journey for them to go on.

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