Impatient for Parkinson's Progress

A drug trial participant struggles to get information

Want to cause an explosion? Just tell someone with Parkinson’s that you have heard there are so many exciting new treatments for the condition these days - why, only last week you were reading about a new drug pump, a new way of getting an early diagnosis, even a wonder drug that could stop the disease in its tracks. You are then very likely to see the Parkinson’s patient lose their rag because they have been hearing about all of these innovations for some time yet, for the most part, their treatment still centres on Levodopa, a drug that was invented more than 60 years ago.

When you have got a degenerative disease like Parkinson’s, the word “patient” is singularly inappropriate - perhaps we should be called “impatients”, so desperate are we for research to bear fruit in time for us to benefit. And if like Alison Elkin you have signed up to participate in that research, then lengthy delays in seeing results can. be even more frustrating.

It was a lengthy and powerful comment posted by Alison on a piece for this newsletter which set me thinking about whether we are justified in being impatient with the slow pace of progress. She started by welcoming the arrival of the new duodopa drug pump but questioning how easy it would be for many people to get it - and, from what I’ve heard about the way many Trusts react to innovations like this she is right to be sceptical.

But her main complaint was about the phase 3 trial of Exenatide, the diabetes drug which could turn out to be the first big leap forward in medication since Levodopa if it is shown to slow the progress of Parkinson’s. Alison said that as a trial participant she was “hugely frustrated” by the “lack of energy and pace in releasing medications for the benefit of Parkinson’s patients.”

So I called her to find out more. A Phase 2 trial held in 2017 had shown results that were promising enough to warrant a much bigger Phase 3 study which got underway in 2020 with 200 participants spread over six centres. Alison was among the very first people to be enrolled, signing up in Oxford just as the Covid pandemic was getting underway.

“I used to turn up at the John Radcliffe hospital,” she remembers. “And they’d thrust this bag at me - I couldn't go in, couldn't breathe over anybody, but they could pass me a bag in the car park so I could carry on with the trial.” She was taking the drug or the placebo - she doesn’t yet know which - for two years and says she’s absolutely certain that her Parkinson’s, first diagnosed in 2016, did not deteriorate in that time. Even after she came off Exenatide in early 2022 nothing changed at first but she says for the last year or more she has seen her symptoms get worse - and she is convinced that the drug was working for her.

Covid did mean the brakes were put on the process of enlisting the participants in the trial and the last people finished taking the drug in early 2024. Alison accepts that the pandemic has inevitably caused delays but she does not understand why the process of adding up the data of 200 people and analysing it is taking so long.

Her trial centre in Oxford had originally said that she would learn in May or June this year whether she had been on Exenatide or the placebo. That didn’t happen, then she was told there would be a newsletter for trial participants in June, then July but the months went by and still no word. She was told that the information would now come centrally from the research team running the trial at University College London but she has found it hard to learn much.

When she has been given reasons for the time it is taking to process the data, she has not been impressed. The fact that one researcher went on maternity leave for a year should not have held things up - “that's highly, highly predictable.” Nor was she convinced, as someone who had a career in IT, that crunching all the numbers was quite the mammoth data processing challenge that it was painted:

“It's not hard to consolidate data from the six sites into one database. I’m struggling to understand why it takes so long, and I just suspect it doesn't need to.”

She says that she doesn’t doubt the professionalism or integrity of the researchers but does wonder whether they understand the urgency of their work:

“Just sometimes I wanted to turn around to them and say - and I didn't - I bit my lip. I just wanted to say, come on, guys, this really matters. This is our lives.”

There is a counter argument. As Alison says, “if this drug does work then that is really massive.” That, however, means the researchers cannot risk rushing things and getting something wrong - that would be a disaster for people with Parkinson’s. But seven years after a successful Phase 2 trial seems more than enough time to get the definitive answer that Phase 3 should provide. And if it isn’t, the 200 trial participants need clearer explanations of what is going on.

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I wanted to put Alison’s complaint to whoever was in charge of the trial but finding that out proved tricky. Eventually, however, a helpful person at one of the funders of the trial Cure Parkinson’s (I’m a patron of the charity) came to my aid. She tracked down Dr Tom Foltynie, Principal Investigator of the Exenatide trial, who provided this statement:

“I fully understand the frustration experienced by Alison in her urgent need to hear news of the results of the Exenatide PD3 trial. We owe her a debt of gratitude alongside all the other participants in the trial. There is absolutely no doubt that the results of this trial must be shared as soon as is practically possible. The data were carefully cleaned to ensure accuracy before analyses could be started at the end of June. The first results were apparent in July. Given the implications of the trial results are potentially so huge, it is of utmost importance that the results are not only accurate, but they are carefully considered and correctly interpreted. We have been working extremely hard ensuring the analyses are complete and have had to do additional unanticipated analyses to ensure we have the best interpretation of the data. We will communicate these as soon as we can. We recognise the importance of communication with participants as well as the PD community in general. Thank you cards are sent/ given when the patients finish the trial. They may not have been approved by REC when Alison completed the trial so there may have been a delay in her receiving this. The last newsletter was sent out in January 2024. Please be reassured that no one is delaying the dissemination of the results unnecessarily. Apologies that this is taking longer than any of us hoped.”

I am grateful to Dr Foltynie for this detailed response. But it should not take an inquiry from a journalist to force a major drugs trial to give participants some explanation about why they must wait so long to hear the results of their efforts.