Balancing hope and hype in Parkinson's research

There’s a lot going on in Parkinson’s research and when it hits the headlines I have learnt to manage my excitement to be positive yet realistic.

Just in the past few weeks we’ve had news stories that the humble appendix, an organ previously thought to be somewhat otiose, may be the very root of the condition itself. This was based on research which identified that in a population of patients who had their appendix removed, there was a statistically significant reduction in the possibility of developing Parkinson’s.

Less accessible for the layman, but perhaps a more significant scientific finding, is the great work being done up in the city of jute, jam, Oor Wullie and serious and impactful medical research, Dundee. Where Miratul Muqit and team, identified the function of the Parkin/ PINK1 genetic pathway as a cause for early onset Parkinson’s. This caused ripples, not just in the Parkinson’s community, but also on the radar of the mainstream media, as it was reported not only in the press, but by the BBC and other high profile media outlets.

Oor Wullie, hiding significant breakthroughs in Parkinson’s research under his pale

There was also the headline grabbing ‘World’s First Pill may stop Parkinson’s’, about an Australian study which had halted neurodegeneration in the ever curable mice, using a compound that modified inflammation. Great I’ll pop down the chemist and collect my prescription…

But hang on a sec… pre-clinical, mice, animal model, may lead to phase 1 clinical trials…So 30+ years away then…

Assuming that it even makes it to phase 1, there are no adverse events in phase 1, there is sufficient funding and robustness of result from phase 1 to merit and pay for a phase 2 clinical trial, that the pill then out performs the placebo in phase 2, there are no harmful side effects identified, the pharmaceutical company considers both the results and the commercial viability worthwhile to fund phase 3, phase 3 also outperforms placebo and there are no adverse events...**pause for breath** and then regulatory approval is granted, the cost is not prohibitive to national health bodies, and lastly that Skynet hasn’t been activated, become self aware, perceived humanity as a threat and proceeded to attempt to eradicate mankind.

Robots, more menacing than they look

There is undoubtedly a lot going on, the three stories I’ve referenced above are indicative of that, and the patient community is correct to take hope and positivity from reading about all of this. However, it is perhaps a difficult message to mange for the individuals and entities involved in research communication and advocacy, just how far away research findings of this nature are from being clinically relevant.

I say this as someone who in my first forays into the world of research advocacy, used to chide Parkinson’s UK for their frequent use of phrases such as ‘one day’ and ‘still much to understand’ in research communications. Whilst I’m still of the opinion that it is important to emphasise the positive impact and sense of progress from news stories such as these, there is also a need to temper with a sense of realism.

So, positive realism…this is how I have come to describe my own attitude to research news such as these, in that I take the positivity from knowing that all of this work is going on, but maintain a sense of realism as to when it will become clinically relevant.

You may ask then, when is it appropriate to get excited by a research news story? I believe it is when there is robust data from a completed phase 2 clinical trial and an announced and actively recruiting phase 3 trial. This is because historically, phase 2 trials, where a small sample of patients are recruited and stratified into active and placebo groups, is where the majority of new drugs have failed. The failure is often due to not achieving the desired impact or perhaps having significant side effects. But after phase 2 trials, it is at that point there is legitimate reason to be hopeful that if phase 3 is successful, the drug/ compound being tested could become clinically available within 10 years or so.

There has been a tendency amongst some clinicians and researchers in the past to quote 5 to 10 years as a time scale of which there will be meaningful impact in the Parkinson’s research field. This has become something of a rolling timescale which resets. And although personally I wasn’t quoted 5 to 10 years upon my own diagnosis, it still resonates with me as I’m about to enter my 5th year under the cloud of a Parkinson’s diagnosis.

I recently attended and presented at a research event hosted by the Scottish Centre for Regenerative Medicine (SCRM) where the topic of timescales was raised. The SCRM receive many patient enquiries, particularly around cell replacement therapy, about when it will become available as a treatment for Parkinson’s. The director of research communications had recently reviewed what they had communicated to patients and their families 10 years ago, about when this would become available, and the quoted time scale was 20 to 30 years. The timescale they quote 10 years later in the present day? Still 20 to 30 years. I quipped at the time given the arithmetic involved, that this should now just be 20 years. The most realistic and perhaps practical answer to such a question is-‘We don’t know’, as setting a time scale perhaps creates expectation that may never be realised.

Becoming an educated patient is key to balancing hope against hype. In the age of social media, where people who otherwise have never connected, are able to network and share knowledge, there are many opportunities to learn and understand both the process of research and what expectations to have from it.

Empowerment through engagement and education is key in many aspects to managing any chronic health condition and part of that is being able to identify when a news story is actually worth getting excited about.

Martin is a Volunteer Research Blogger at Parkinson’s UK, he writes perspective pieces about Parkinson’s research. Read other blog posts by Martin