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11th of April 2019

Benjamin’s quest

Pushing forward to connect the dots of Parkinson’s

The pace of discovery is slower than the pace in which the neurons in my brain are dying. That is the simple truth. We have to have the courage to face this | Ben Stecher (1)

Benjamin Stecher is a Parkinson’s advocate who was diagnosed in 2013 with Parkinson’s at the age of 29. At the time he was living and working in China as an education consultant. Education has always been very important to him and after his diagnosis he felt that he had two choices: Either he could go to medical school and get a PhD or he could start finding the greatest minds in the field, reach out to these experts, interview them and start connecting the dots.

Benjamin chose option two and in 2016 he started a blog called Tomorrow Edition. Since then he has interviewed over 100 experts and his teachers have given him a unique perspective on Parkinson’s. A perspective which he shares on conferences and events, such as a 4 hour seminar he delivered (1,2) at McGill University in March, 2019.

In this post I distill four ‘grand questions’ from Ben’s talk which need collective answering.

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Question I: How can we educate Parkinson’s patients to deal with their disease?

When Ben got his diagnosis, he visited a Chinese doctor who strapped poppy seed to his ear and told him to walk West every day for one hour. Ben didn’t run the experiment, but states this was just one of the many rabbit holes he had to fall through. According to Ben not a week goes by without someone mailing him about some new therapy, exotic formula or strange location which might cure the disease. Ben thinks that Parkinson’s patients can prevent themselves from falling down the same rabbit holes again and again by educating themselves in what to believe and what not to believe.

The average Parkinson’s patient gets one hour a year with the neurologist and 8765 with self care (3). To help Parkinson’s patients manage their disease on a day to day basis, Ben thinks more information should be made available to try and understand the optimal lifestyle. For example, how does sleep, anxiety, the amount of water we drink and the exercise we take influence the way our medicine is absorbed?

Science has an important role to help patients understand what is true and Ben calls upon all scientists to deliver their knowledge in easily digestable chunks. Ben also thinks that more elaborate education programs should be made available to patients. Programs where they can learn about the disease and have an opportunity to talk to researchers.

Question 2: Can we hold science accountable to patients?

Ultimately the question is: for whom do we want to solve the disease? | Ben Stecher  

Benjamin observes that a lot of science seems to be done for the scientists themselves (or their funders), not for the greater good. In spite of efforts in speeding up discovery through open science (4), a serious gap between the research community and the patient community exists. The patient’s voice is absent from the discussion way too often. For example, Ben has visited lots of biotech companies where he was the very first patient they ever laid eyes on. Such an emotional distance can be helpful in some ways, but in the end this doesn’t help to stay motivated for the right thing: working towards understanding, halting and reverting Parkinson’s. Patient participation is all about bringing the larger picture to live again. Great research does start from ‘Why’.

If we want research to be more effective in helping to solve the problems our world currently faces, we have to be better | John Tennant, introducing the Open Science MOOC

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Question 3: How can we embed complexity in our research endeavours? 

Nature does not work in what we perceive as a logical sequence of causes and effects. This creates a lot of mistaken beliefs about the nature of complex diseases | Alberto Espay, Interview by Ben Stecher (5) 

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How does Parkinson’s start, why do dopamine neurons break down, how does Parkinson’s progress? In spite of our efforts, we do not yet understand the basic biology of Parkinson’s. Therefore, ASAP thinks we need to assume we know nothing and move forward in understanding the basics from there.

At this moment science mostly works in a reductionist approach: we isolate a variable and investigate one target at a time. That is simply the way our minds work. We understand the world as a sequential series of events. One thing happens, then another thing happens, etc. A lot of people think of Parkinson’s as a puzzle, but that picture is misleading. We may be looking at pieces of a puzzle, but the pieces may all belong to different puzzles. How much complexity do we need to incorporate in our study of Parkinson’s? And how can we keep on challenging existing beliefs and prevent ourselves from falling down the wrong rabbit holes?

Our minds love stories. That is what makes us human. But that doesn’t make the stories true.

Benjamin puts forward an interesting thought experiment. What if we would stop focusing on one target in a large group of patients (as we do in clinical trials) and instead put all of our focus on outliers: those people who make substantial progress on reversing their symptoms and take them as our object of study?

Question 4. How can we make clinical trials more efficient? 

At the moment there are 286 clinical trials on Parkinson’s disease ongoing (6).  This is encouraging, but according to Ben most of these trials aren’t going anywhere. Clinical trials have a problem with the ‘Parkinson’s is a single disease script’. At the moment, clinical trials enroll people with Parkinson’s in one broad category and try to treat them as if they have one disease. But they don’t.

Benjamin has co-authored an insightful article (7) which makes clear that it isn’t only the type of Parkinson’s which matters but also the stage of the disease. Patients which have already developed serious symptoms, may be past the phase where they can be cured with medication which focuses on an earlier event in time.

Also, the current outcome measures of clinical trials are poor. With the UPDRS you have to do laboratory tests which you never do in real life. The thermometer measures the wrong things (8).

These pitfalls unavoidably lead to failure. Currently, if only a subset of people benefit from a drug, a clinical trail will be cancelled and the underlying data are often never exposed. But couldn’t we learn more about the disease from such a subset of people than from pushing forward a cure for the ‘average person with PD’ which doesn’t exist?

Benjamin does think we should move forward with clinical trials, e.g. with the Linked Clinical Trials initiative (LCT) for Parkinson’s disease (9). LCT is a drug repurposing programme specifically aimed at identifying drugs that can slow the progression of Parkinson’s disease. In the end, the only complex enough model of Parkinson’s is people having the disease. And having a chance of succes is better than having no chance at all, Ben thinks.

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Wrapping up: Why is Ben’s work important to me? 

Ben thinks the patient’s voice should be included at every level of decision making. And I couldn’t agree more. His ‘grand questions’ show that patients are in a supreme position to catalyze the connection of some Parkinsonian dots. The main reason being that patients serve as a living example of keeping the WHY in Parkinson’s research alive. Because where a scientist may be able to escape the why, a patient simply cannot.

I admire Benjamin’s way to look for new connections to speed up discovery. All of the individual researchers he interviews at Tomorrow Edition seem to be playing their instruments very well, but in the end we do need conductors who get all players together. Conductors who remember scientists about the fact that they are in fact playing in the same choir and have to adjust their ramblings accordingly.

I’m truly looking forward to a symphony in PD.

Sparks

Sources

(1) Stecher, B. Tackling Parkinson’s Disease (March 25, 2019). [Youtube Video]. Retrieved from https://youtu.be/VLyv2xoa0VM

(2) Hayward, S. Listening to the patient (April 1, 2019). Parkinson’s advocate says patients have a greater role to play in research. [Blogpost]. Retrieved from https://www.mcgill.ca/neuro/article/patient-stories-research-stories/listening-patient

(3) Riggare, S. 1 vs 8,765. [Blogpost]. Retrieved from http://www.riggare.se/1-vs-8765/ 

(4). Verbeeldingskr8 (2016). Open Science: New models for opening up research outputs. [YouTube Video]. Retrieved from https://youtu.be/4Y_IOea6ijU 

(5) Stecher, B. (January 22, 2019). Interview with systems neurology expert prof. Alberto Espay. [Blogpost]. Retrieved from https://tmrwedition.com/2019/01/22/interview-with-systems-neurology-expert-prof-alberto-espay/

(6) McFarthing, K. (April 3, 2019). The hope list – Parkinson’s therapies in development. [Google Docs]. Retrieved from  https://drive.google.com/file/d/1NeSyFA37b9IbUzryRRP-EqrgScjCRL-3/view

(7). Johnson, M. E. , Stecher, B. , Labrie, V., Brundin, L., Brundin, P., (2018),  Triggers, Facilitators, and Aggravators: Redefining Parkinson’s Disease Pathogenesis, Trends in Neurosciences, Retrieved from https://doi.org/10.1016/j.tins.2018.09.007 

(8) The Ben and Bas show. (April 6, 2019). [Youtube Video]. Retrieved from https://youtu.be/Gj1iXHUWhSk

(9) Brundin, P., Wyse, R.K. (2019) The Linked Clinical Trials initiative (LCT) for Parkinson’s disease. European Journal of Neuroscience. Volume 49, issue 3, pages 307-315. Retrieved from https://doi.org/10.1111/ejn.14175

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