If it wasn’t for the expectation…

Back when I was a child chocolate was an education, learning all those long words, as well as a treat, and I am pleased to say, now that I am an older adult, internet research uncovered evidence that dark chocolate is good for you. I like it when things have than one positive outcome.

Posts on the Science of Parkinsons blog not only provide useful information but sometimes set me thinking.  A recent post An exercise in expectations – Exenatide III set me thinking so hard it made my head spin.  I needed to sort out my own thoughts and since writing helps to clarify them, in desperation, I have put fingers to keyboard in response.  Not many people read my blog but maybe some will and will have helpful comments to contribute and if not at least I’ll have got my own thoughts in order. The SoP post set outs the argument for avoiding raising expectations and keeping an open mind, I am not disagreeing, but maybe its only part of the story.

Let me introduce some pacification right from the start; I completely agree that the clever researchers into any currently incurable conditions need to keep an open, unbiased approach and just let the science do its work.  Being unbiased is hard enough at the best of times. However, when you have devoted many years of hard thinking and work to a particular path of investigation, to have to contemplate that a result that might be expressed, and I simplify here, as “Listen up colleagues/funders, we’ve been successful in showing this drug doesn’t work” must be hard.  It takes a level of objectivity that seems totally unsympathetic to patients. But acclamation of the work is necessary if the researcher is to follow the path to more funding for the next project, which might be “the one”.  More than that it’s a perfectly valid and important research outcome, if one that is hard to hear by those living with whatever desperate ailment is being studied.

In recent years the Parkinson’s world has moved towards a much greater involvement of patients in research design, prioritising research directions and raising the profile of Parkinson’s research.  There is a developing cast of Parkinson’s Advocates that are very visible on social media, talk at world conferences and are known personally to leading researchers.  I think this is a good thing but it must make it even harder for researchers to take a step back and maintain the objective distance required in the face of real lived experience.  I would guess that spending 3 days with a person living with the consequences of Parkinson’s at a conference is a bit different to the usual half hour consultation.

I also agree that the broader Parkinson’s world needs to manage the expectations of the millions living with the condition.  It’s a tricky balance between raising awareness of the need for a cure and better treatments, which is likely to drive greater investment in research funds and overegging the potential and thus disappointing patients, but it’s a balance that is important.

Where the whole thing gets a lot more “difficult” is with patients, particularly those who might volunteer as research subjects

“Honestly, clinical trials for Parkinson’s would be so much easier if we could just take the humans out of them.”, says Dr Simon Stott author of Science of Parkinson’s. 

I am sure he’s right there but, for the time being, we’re stuck with the gold standard randomised, double blind placebo trial on humans as a vital phase of bringing any new or newly purposed drug into use. 

So what are the problems with patients?  Let’s start with expectations?

Using a thesaurus to look for synonyms for expectation includes anticipation, promise and significantly hope.  It is the hope of avoiding an unpleasant end to life which is represented by finding a cure that keeps many living with Parkinson’s going and keeps them, and their family and friends, actively raising money for research and volunteering to be research subjects.  I think there has to be a bit of a bias toward positive expectations to fulfil a need.

Now let’s look at those who volunteer as research participants. 

As a basic principle why would one volunteer to take part in a drug trial if you didn’t have a reasonable expectation that this treatment was going to work.  I have huge admiration and respect for the brave and positive individuals who took part in the GDNF trial, but I suspect that all the counselling and managing of expectations in the world wouldn’t have quelled that kernel of hope deep inside that the treatment was going to work.  Was it that hope that gave them the strength to go through with the trial?  Thinking about the Exenatide phase 3 trial, as I understand it, participants have to inject themselves weekly for 2 years.  Now that’s a tough ask when you consider that the outcome might be that it has no effect.  But when there is a 50% chance that you are injecting a placebo, so even if there was a positive outcome you won’t have benefitted it’s an even tougher ask.  To take part in that sort of trial takes a mind-set that already sets people apart from the norm.

I am not familiar with all of the ways that patients are recruited for trials.  I do know that it’s not always easy to recruit and that some research fails to get off the ground as a result.  Several web based matching projects have been developed in order to encourage participation in Parkinson’s research.  I am linked in to a couple myself and have taken part in 3 trials so far, all observational.  I suspect that those that get involved are more likely to be people like me who are interested in the condition and spend time trying to understand it. Not as bold maybe as those prepared to try drug trials, but still not the norm.

So far a biased sample.

As I often say to people “One wouldn’t choose to have Parkinson’s but at least it’s interesting.”  It’s actually fascinating with all its complexity, different types of symptoms and the different approaches to try to manage different aspects.  I spend quite a bit of time reading and researching and trying to put into practice ideas that seem to make sense to me for my own practice personally and sometimes for the wider community. (See earlier post for example) For myself, adopting the “It can’t do no ‘arm if it don’t do no good” approach, and inspired by the activities undertaken in a particular study I have added saccadic eye movement and eyes closed balance practice to my ever expanding exercise regime.  I am always alert to including different exercises, physical and mental, into the list.  Am I making a difference? It feels like it, I can’t prove it, but it does help me to feel more positive about being in control and that definitely does make a difference, and that’s enough realisation for me.  But here’s a dilemma, if I were to take part in a long treatment trial, would I be potentially spoiling the results by implementing other strategies for making a difference, that I want to try, while the trial was in progress? I’m sure I am not unique in my approach. I wouldn’t want to wait 2 years to try out a new activity, exercise or medication suggested by my clinician.

My conclusions – while patients, with their hard to live with symptoms and their ab”norm”al perseverance, attitudes and strategies are involved in trials, expectations are going to be high, samples are going to be biased and subjects might skew results.  Tricky isn’t it? Difficult for researchers and patients.

Getting to the satisfaction of eating the chocolate, is lost in the depths of time.

image source pocketbookuk.com

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