It is one of the questions I get asked the most from the research community. What could I as a lowly scientist be working on to help people living with Parkinson’s diseases today?
Well, that was exactly the question I sought to answer in this talk given at the EPFL just two weeks ago…
(For now I will just upload the slides from that talk here but the video should be coming soon, right Hilal? 😉
However, I have since realized that might not be enough as those are more general principles than practical steps. So, here is an experiment that I believe we could start doing today and that I would love for somebody to run with:
The TESOBO Experiment
(Tackling Each Symptom, One By One)
My thinking on this started with a relatively simple question that there seems to be no answer to – How many different symptoms are associated with the label clinicians refer to as Parkinson’s Disease?
However, the more difficult yet far more important questions are: How many different causes are there of each symptom? And at what point does any one symptom emerge from any one cause?
The answer seems unsettling as for any one symptom there are dozens of potential causes. How can we ever root out each? Well, while that would be nice, I think many patients would rather researchers focus on figuring out what we can do now to better control each.
So, here’s a possible experiment a researcher could take upon themselves to begin doing now that just might help:
Start with a relatively simple yet classical symptom of these diseases. Make sure it is something we can now empirically measure, like tremor. Take 10-20 tremor dominant patients, hook them into Rune Labs‘ StrivePD and observe them for 2 weeks monitoring everything possible. (That means what and when they eat, sleep, drink, go to the bathroom, brush their teeth…everything! While collecting every fluid, biosample and potential digital diagnostic marker possible.)
Important notes: Be unbiased and open, meaning let data guide discovery and publish everything as soon as reasonably possible in an open document. Also, pay participants, which is self-explanatory, I hope. And involve people with these diseases in the design and implementation of this study, use their expertise to help you decide what parameters to focus on, what data to collect, how to collect them, how to interpret the data, who gets to use that data and for what, as well as how to disseminate it all and what the next steps should be. (Thanks Dr. Sara Riggare for reminding me to include that last sentence!)
If done right we could start to figure out what works and what doesn’t for some individuals with tremor. We would then need to expand the cohort and the length of time to start being able to approach statistical significance. But, the point is that this experiment is doable, and should be done.
However, it is important to note that no matter how we design this, we will fall short of the pie-in-the-sky goal of most research today, which to relieve these symptoms forever. And, as you’ll see in the chart below, tremor isn’t even one of the problems people living with Parkinson’s think of as the symptom that challenges them the most.
The good news is though, progress is possible! We can apply the same methodology described above to some of the things patient’s themselves list as the most bothersome symptoms they experience. Some can be empirically measured with digital devices, others require more patient reported outcome data, but there are only a few things in the list above that cannot be measured at all.
So researchers, hope that helps answer the question posed above. Please feel free to post any follow up questions you might have. While we are here, here are a few for you to ponder:
- How big should the initial N of people be for the scientific community to be able to glean anything noteworthy from this particular fishing expedition?
- Do you know of any labs that are or might be interested in working on this?
- And of course, who might fund this (or something like it?)
(Thanks Dr. Nora Bengoa-Vergniory for helping me think through those questions!)
For those diagnosed, please let me know if you think I got anything wrong here or if there is anything else you think researchers could do to help you today just write it in the comments below…
Here, here Ben. A study where everyone involved is both participant and observer.