Patient defned PD
 

Harley got me to thinking about how I would slice-and-dice the PD pie. It is not a monolith, but what IS it then?

We differ on symptom sets, speed of degeneration, present state, body systems impacted, and more I'm sure.

Maybe a matrix of some sort with disease features across the top and the impact upon quality of life down the side? In fact, make QOL the feature reflected at any given time? Does that make sense?

At least I see why they don't want to discuss it. :D

a puzzle
 

Rick - the puzzle is further complicated and the matrix expanded by the addition of the myriad of treatments available, the countless combinations of those treatments, and the endless individual responses to those treatments. Our QOL is not only impacted by symptoms, but how successfully we can control those symptoms. Two people may have the same amount and placement of tremor, and one can treat it successfully, the other not at all. And what portion of our symptoms are actually a product of our treatments? Hard to chart, eh??!!

But for research, this extreme variation might also be the point. Regardless, patients hold the key.

I wonder if we should concentrate on the things we all share? What are the pieces of Parkinson's we all have in common? Slowness seems to be one commonality - of movement and thinking.

I think I know the answer!
 

I have really been giving this a lot of thought lately, and it has to be one reason we cannot find better treatments, and (surprise!) it has to do with the placebo effect.

The placebo effect is why most of the major studies have failed.
Animal models will show us very little other than when something is toxic. Why? Animals do not get Parkinson's, AND animal model studies do not have placebo effect. I think it is as clear as the nose on one's face that the executive functioning and emotional reactions of humans to any of these therapies are why trials fail - we just aren't measuring that simultaneously with motor symptom outcome measures.

Why can't there be a scale or at the very least a form used consistantly in some of these trials to indicate when a person is going through emotional issues. And a complete neuropsychiatric evaluation should initially be done at the onset of the trial and again prior to looking at endpoints. A friend and I are doing an informal record of anecdotal notes to see if there is a connection between how we "feel" and when our dyskinesia increases. I have already placed a wager that when we are upset, motor symptoms exacerbate.

I realize that this would be extremely difficult to get anything other tha n subjective inpput results, but if we can but show the associaton, then we will be onto something.

Now, I would like to hear what others think about this? Maybe I am unaware of trials that used this approach. If so, I would love to look at some of the findings.

Peggy

Some good points
 

I've long suspected that a key was hidden in the placebo effect. As a group, we seem to have the strongest of any.

Can we put together a core group of symptoms hared by at least 75% of PWP as a starting point. The obvious ones and then think about the next step. Wait a minute though. Time is a factor. For example a hypersensitivity to emotional distress is near universal in Year 10 of PD, but often non-evident in Year 1. Maybe once we have the core list, the next step could workon that.

So, without factoring in time at this point, here are some that I think 75% or more of PWP experience at some point in the course.

Exaggerated response to distress.
Rigidity.
Slowness of movement.


Here are some that I think 50% to 75% experience-

Tremor
Gait problems
Balance problems
Cognitive problems

And here, the 25% to 50% group.

Depression
Sleep problems
Dystonia

And, finally, the less than 25% group.

Skin problems


Remember, this initial list is talking about what PWPs who live out a course of PD will experience over the entire period. If you live with it for forty years from date of first symptoms (a good origin for a baseline so long as we allow negative numbers for pre-clinical conditions), you are over 75% likely to experience hypersensitivity to distress at some point. But you wil probably have good skin! :D

Try adding some more and point out ones that need a different frequency. Then we can figure out what to do next.

Does anyone have any of the surveys of symptoms?

Diy
 

why dont we create our owm, surveys were the front line players.same old questions archieved data on forums,there is an incredible pool of people here. patients are leaders,there potential as yet unrealised

Thinking the same thing
 

I was thinking the same thing. I'd be curious to see how many others experience the something like a periodic paralysis like Rick and I do. I can sign up at Survey Monkey and the quiz can be take anonymously through a link. It's very easy to get the survey up and running. I'll do that part if we want to start listing some symptoms and questions.

Laura

question?
 

How would you know if a symptom is PD connected or something else if you have other health problems?

When I first started with the hand tremor I thought it was connected to the breast cancer or treatment but found it wasn't. My mom died of ovarian cancer which went undetected because they didn't run the correct tests and thought it was just a problem she had with IBS.

Sorry about your mother. It happens all too frequently.

As for your question, my guess is you don't know - if you think PD causes a symptom, toss it into the ring and if no one else of bejillion PWP mentions it, it's probably not Parkinsons. Or you are just unique - as we all are.

Is this the kind of thing people are thinking of doing?

Observational trials -- ~20,000 people needed!
 

I just commented in thread #136157 -- how does one "cure" parkinson's ... so some of this is redundant.

Yes scientists appreciate this random mix of extensive motor and non-motor symptoms in PD...and, they are beginning to better understand the pacing of such symptoms. There are extraordinary challenges in observing, collecting, analyzing and generalizing such data. But, rest assured much of this work is being attempted (and funded by MJFF).

It might surprise folks to know that right now there are observational trials open and recruiting in the US that seek nearly 20,000 participants (mostly PD patients and some controls too). In these observational studies, researchers are trying to observe and follow these complex experiences in patients so we can not only better understand what PD really is (and isn't) but importantly so we can be best informed as we prioritize research, identify clinical populations and try to measure efficacy of treatments.

Observational studies (and I admit, not all are created equal!) are a great way for patients to teach scientists. There are scores of questions that researchers have posed or observations they want to validate that depend on you for answers.

Please education yourself and/or ask your physician about observational studies (well, of course interventional ones too!) where you can contribute to accelerating knowledge and progress.

I appreciate that even across all the current studies there may not be one that is asking the specific questions that you think are important. Asking questions and gathering reams of data is expensive and daunting. But, I am a believer that the patient community can rise up and show that researchers that they want to participate in these types of teachings, more and more probing and relevant studies will be designed.

Debi

PS...MJFF is developing various tools and programs to help address the challenge of poor participation in trials and looks forward to more input from patients on this front.

A Story
 

I often send emails to Debi with one concern or another, sometimes big, sometimes small. I know she pays attention, replies often - but listens and takes note always.

A couple of weeks ago, I sent her an email with a request for a phone conversation regarding possibilities for an expanded patient voice / role in clinical studies and research in general. And, mostly, to talk about my "global worries' that the gains we have made in the past 10 years - namely, that it finally seems that the "important" people - researchers, organization personnel - have a good understanding that they don't fully understand Parkinson's, and that the patient experience with Parkinson's should be carefully studied in order for the research to continue in a successful and relevant way (hence Debi's call for our participation in observational studies - they are crucial!).

I was also acting on the energy that was coming from patient comments on their WPC experiences.

The result was a wide-ranging, frank, informative conference call yesterday that included Debi, Laura Dalle Pazze (MJFF point person on clinical trials), Kate Peabody (Special Assistant to Debi), Greg and AJ Wasson, and myself.

The conversation ended up being almost two hours in length - thank you! Debi patiently and exhaustively answered all of our questions, fielded all of our suggestions, discussed many possibilities, and pointed out several inconvenient truths. She left me feeling overwhelmed and optimistic. The realities of the drug development process remain frustrating (financial, regulatory, historical) - some things we need to confront and change; others might be set in concrete; all are not easy to challenge, but can't be ignored.

I do have no doubt that MJFF understands our every concern about the mysterious ways of Parkinson's with all of its many manifestations - it stymies, frustrates, and impedes their progress as well. PPMI will hopefully provide some answers. And, again, observational studies are designed to help fill these gaps. I also know that, when it is in their power, MJFF insists that trial protocols be designed with the patients' best interest in mind (that potential discoveries be made available to trial participants; that endpoints can flexibly react to developments during the trial; that the pool of participants reflect - as best as can be expected at this point in time - people who actually have PD, optimum transparency, etc).

Our third MJFF Patient Advisory Council meeting is next month in NYC. Again we will have the opportunity to interact with the MJFF Scientific Advisory Council - and I look forward to talking with MJFF staff in person and following up on this conversation.

Bottom line - MJFF listens to patients (you all know they read here), is incorporating us in their programs and as part of every process, values our experience, and understands our frustrations - and why we have them. I still worry - gotta keep vigilant - but I know I am not worrying alone - we have some pretty powerful people who share and champion our concerns.

Thanks
 

Thank you, Carey. And thank you, Debi.

Jean

A modest suggestion
 

Debi-
Why not have someone, maybe a grad student even, whose job is to go back through the forum archives to compile one or more reports on what the research community ought to know? It is a shame that so much of what we speculate on is never given a chance to catch the interest of someone who could follow up on the good stuff.b

Observation Studies
 

I have the ones not recruiting hidden if you're interested viewing them -unhide them.

i think i'll check out #29

http://clinicaltrials.gov/ct2/result...ease&recr=Open