1. have to verify someone has pd? that they are honestly reporting data?
2. who's going to verify that the data is being stored and analyzed correctly? not saying someone is going to lie but mistakes happen.
3. you can't expect most people to wait when "Off", then take the drug, then wait for the affect, think about it. so how are you going to decide on dosage amounts, times, when to report?
4. most clinical trials require doctor approval, are you going to take responsibility for dire consequences?

you've got drug companies spending millions designing trials and analyzing the data using highly trained professionals. this isn't cancer or diabetes or treating high blood pressure where it's easy to take measurements, your're measuring small changes over time in a patient where their pd changes from minute to minute, that requires some knowledge of biology, statistics, science.

so lets say you try a ginseng trial, how many patients will you need and how much of a difference will you need in finger tapping over a control to say it has a significant affect at the 5% confidence level? do you have a normal population sample? can you verify someone took the ginseng? how long do you do the trial?

i always play devil's advocate, i suggest you just propose a trial, say take ginseng, get people to check blood pressure (is it safe) , etc., and then just have people keep a diary and report back in 6 weeks. if you can organize 10 people to do that, then build on that. if you can't, then figure out why, it's difficult enough to recruit pd'ers for regular trials.

i can finger tap differently every hour even when my sinemet is working well so i would think you need more measurements than that, i can also be in a terrible brain fog and finger tap well.

How to get valid information from "amateur clinical trials"?....... I have a few suggestions and comments.

1. have to verify someone has pd? that they are honestly reporting data?

There is no way to be absolutely certain that someone has PD unless you see that person. Inclusion criteria will have to be based on trust and assumption that people who post here and participate in any given study care about getting better and want to have a PD-free life. It is not very scientific, I agree.

For starters, a questionnaire about age, year of diagnosis, symptoms at the time of diagnosis, current symptoms and medications would give some structure to the study. If people are willing, one could do a Skype meeting to confirm PD.

One major assumption is that they are reporting data accurately and honestly. All I can say is that I trust my fellow PwPs.


2. who's going to verify that the data is being stored and analyzed correctly? not saying someone is going to lie but mistakes happen.

In this aspect, I do not see much difference between "amateur clinical trials" and professional clinical trials. Data collected will be with the "PI" (Patient Investigator) just as the "PI" (Principle Investigator) of a clinical program does. Since amateur data will be available to public, it can be analyzed by any patient who has expertise and/or interest in the study, similar to peer review process of a scientific community. I bet we have researchers, clinicians, statisticians etc., among us (who can help....).

To take this process one step further, amateur clinical trial data can be presented in a PD meeting and if the study yields interesting conclusions, there would be even more scrutiny of the data. Since PwPs have no career or financial interests at stake, negative data might even come out!

3. you can't expect most people to wait when "Off", then take the drug, then wait for the affect, think about it. so how are you going to decide on dosage amounts, times, when to report?

These issues can be resolved with detailed instructions, good record keeping and detailed protocols for how to take a finger tapping test or any other tests for PD. Obviously we cannot mess with dosage of meds, times etc.,

We may not be able to pool data from, say all the 100 participants to get a conclusion because of variations in drugs, dosage etc., But we can always measure the improvement over baseline within a given time for each participant. for example, I measure my finger tapping rate after 30min of meds (whatever meds and how many times I take is recorded) for a week or two before I start on a new suppliment. Keep records for 6 months with the new compound. The key is to maintain detailed records. If needed, participants can exchange info in a skype meeting once a month or so.

4. most clinical trials require doctor approval, are you going to take responsibility for dire consequences?

No, unfortunately these patient-initiated trials will be no different than professional clincal trials in terms of taking responsibility for dire consequences! Inclusion criteria: Lab rats who understand "Informed Consent" concept and do not blame others for their deeds.

you've got drug companies spending millions designing trials and analyzing the data using highly trained professionals. this isn't cancer or diabetes or treating high blood pressure where it's easy to take measurements, your're measuring small changes over time in a patient where their pd changes from minute to minute, that requires some knowledge of biology, statistics, science.

Whether it is easy or not, drug companies are not interested in studies we are talking about. And also, we need to start at some point and improvise as we go along. Cannot wait for perfect conditions to test our hypotheses.


i always play devil's advocate, i suggest you just propose a trial, say take ginseng, get people to check blood pressure (is it safe) , etc., and then just have people keep a diary and report back in 6 weeks.


Agreed!


PS: I prefer the term patient-initiated clinical trial rather than amateur clinical trials

Girija

i guess my point is if not done right at the start people will lose interest and you won't know if your results are really valid. so i disagree that you just need to get started and work out the bugs later, that is amateurish research. and if the benefits are so great that you can do sloppy design why isn't everyone taking that supplement/drug already?

there's already websites that report patient opinions about what they are taking, does anyone with pd take what is posted seriously?
http://www.patientslikeme.com/condit...nson-s-disease

here's a simple trial, see what the affect of taking your first dose of carbidopa/levodopa with 8oz. of orange juice or hot water with 1tsp of sugar dissolved to test gastric emptying. if you are using finger tapping,record 10 days without orange juice and 10 days with orange juice. that's something everyone taking sinemet or stalvo could try.

so the question is how long after taking your meds do you start finger tapping and at what time intervals? does the orange juice make you go on quicker, does your on "last" longer? so what if you find out you go on quicker but go off sooner? even this simple test can result in complex results, and just saying you can tap faster 30min after taking your meds might not make the treatment useful.

soccertese,

In an ideal world all research would be professionally run to the highest standards. In an ideal world, there would be well researched answers to everything that we're interested in from grapefruit to ginseng, from coconut oil to forced exercise, from curcumin to milk. In an ideal world, the information would be with us now. Unfortunately, we don't live in an ideal world.

In an ideal world, we would be able to put off making a decision until we had full and accurate information. Unfortunately, we don't live in an ideal world.

When it comes to trying to reduce our symptoms or to slow the rate at which our PD progresses we are forced to make decisions now even though we have limited information.

For instance, I'm interested in curcumin. Do you think I should wait until an "official" trial is completed?

We get anecdotal information from this forum. For which I'm very grateful.

But the gulf between this and "official" information sources is vast. So, the question is: can we do better?

I believe we can in three ways:
- we can aggregate the experiences of people (even two reports are better than one);
- we can measure the effect of an action.
- we can open the data to anyone to mine.

Is this an ideal world solution? No.

Is it a reasonable response to a real world problem? Yes.

On specific points in your post. I'm not proposing that people enter a clinical trial. Rather, I see people doing whatever they normally would do. The only difference is that they report the result of their actions. Of course, people will take different amounts of a supplement, at different times and for different periods. This leads to "dirty" data and the need for data mining.

You mention finger tapping not telling the whole story. You are correct. Are you running my program? In a similar set-up to what I run, Vokear et al. [1] measured "Upper limb motor function" using "the finger tapping test [in which] Subjects had to hit two buttons separated by 30 cm alternately during a 30 second period." They found that in the off state this alone explains 58% of the variance in UPDRS III scores.

Again it comes down to whether to wait for an ideal world solution or to proceed pragmatically.

I do hope you send us your data.

Reference

[1] "Effects of levodopa on upper limb mobility and gait in Parkinson’s disease"
M Vokaer, N Abou Azar, D Zegers de Beyl
J Neurol Neurosurg Psychiatry 2003;74:1304–1307
http://www.ncbi.nlm.nih.gov/pmc/arti...v074p01304.pdf

John

"For instance, I'm interested in curcumin. Do you think I should wait until an "official" trial is completed?"

let's not be ridiculous. that's not what i'm implying. if you've read my posts you would know i've tried about every supplement out there, far more than you have i suspect and i have been taking curcumin for years, can't say i see any overt benefit but maybe it's neuroprotective so i take it. i've tried low dose naltrexone, i.v. glutathione, chelation, etc., etc., etc.

you really didn't read my last post, no point in repeating myself.

soccertese,

You write "you really didn't read my last post". Of course I hadn't when I submitted my post. If you had checked, you would have seen that my post was just 4 minutes after yours. So, I was writing my reply to your original post when you made your later post.

You write "i've tried about every supplement out there, far more than you have i suspect". I'm sure that you're right: I've probably taken supplements on fewer than 50 days since being diagnosed nearly 8 years ago. Though whether taking supplements is a good thing is another matter. It just goes to show that all of us act on limited data, and all of us have an interest in better quality data.

Your idea of measuring the impact of orange juice on Sinemet is a good one. I look forward to reading your results. You ask:
"so the question is how long after taking your meds do you start finger tapping and at what time intervals?"
I would advise starting the test at least 30 minutes BEFORE taking the meds. This let's you form a baseline. I would recommend time intervals of 10 minutes, this allows the area under the curve (AUC) to be estimated. The outcomes are indeed complicated, but if I had to choose one metric, it would be AUC.

I'm glad that you now see the benefits that come from PwP measuring their symptoms.

John

Just some other possibilities...

How to tell some one has PD? Google SWEDD. No one can tell what PD is let alone if we have it. We have no scientific basis to tell that even upon autopsy. There is no proof that patient driven data is any more erroneous than clinical diagnosis.

As to the tapping test, this is the only type of measurement that science and medicine currently offer neurologists, so I do not how that is inherently better than anything patients might come up with.

Laura

There are many things I have personally noted about the lack of "science" in our scientific research, but will just point to the experts:

Please consider this info and see if you still have the same concerns.

Based on the meager results page of a Google search, it seems that not too many people know of Dr. John Ioannidis? He wrote a landmark paper in 2005:

Why Most Published Research Findings Are False.

This is a patient friendlier read in The Atlantic Monthly: Lies, Damned Lies, and Medical Science

Even his critics at Johns Hopkins contend that he is basically correct.

I think that PD research fails on many of his points. I don't mean any disrespect for researchers; they are caught up in a "system" just as much as we are. However, I don't think we can sit back and say they have absolute authority on asking questions, seeking answers, and sharing information. What John and Rick are proposing is not nearly as harmful as an unproven hypothesis becoming the core of research for over 50 years. Maybe this is a factor in why we have no cure?

I will say maybe for legal reasons we need to rethink the rat lab as a source for a group name. 23andme is calling those in PD cohort "Citizen Scientist" what if we took on name "Patient Scientist" or "Impatient Patient" tee hee

Laura

laura,
drug companies might purposely design trials that make their new drug look better than an existing mediocre drug but that it totally different than a poorly designed trial that as a result doesn't actually test your hypothesis.

that said, there sure have been a lot of failed pd trials and drug trials in general that have cost billions and billions of dollars.

as far as finger tapping, i'd say if you want to test that as a measurement, measure your tapping before your first morning meds and at 15min increments after 1st meds up to 1.5 hrs for 5 days and see if there is much of a difference.

I think any trial, patient-designed or other, is fatally compromised before it starts because we still don't know what Parkinson's is. Any trial cohort is diluted with people who don't have PD, or have different manifestations of PD.

Until we get some identifiable biomarkers, all else is speculation - influenced by the too-hard-to-control placebo effect (especially by those who are testing their own reaction to pills taken with orange juice).

If we have learned anything from taking sinemet for so many years, it is that symptom control has little to do with halting progression or curing the disease.

A fix for Parkinson's lies beneath the surface; taping before and after downing our pills with OJ will tell us only what we want, or don't want, to believe.