I understand and agree that the methodology for evaluating PD is not good at t his point, no biomarkers, placebo effect, not even a clear definitiion of what PD is. These are the reasons many clinical trials failed.

But does it mean, we as patients need to wait for ideal conditions to check out what makes us feel better? Like John and Rick stated why not just document what we have and try to make it a little more scientific. We are not conducting major clinical trials here to cure PD. Just testing if what worked for Rick would work for a few more PwPs and document those findings the best way we can.

Yes. There are going to be false positives, placebo effects in our experiments. after all, placebo effect is due to dopamine release in the brain. Isn;t that we all want and need? As a patient, I would take a placebo effect if it makes my life a little better.

I have seen and been a part of the research world and I can say that very few scientists feel the sense of urgency for a treatment as a patient would. No one is interested in checking out if medication works better with OJ or pepsi. There are no funds for such work.
IF we want to feel better at this moment, however small that might be, we need to do our own experiments, at least those who are willing to be lab rats/
Girija

not to get stuck in minutia, but i mentioned that orange juice + sinemet not because i think it will actually be beneficial but just as a "dry run" for measuring data, would it actually show a difference and if so, would you actually feel "better". just a trial run to see if you could get volunteers and how the data collection worked out before attempting anything more complicated.

that's an excellent point that we are all different, strong placebo affect, etc., but at the minimum i can see having a testing/data collection system in place that we could use for just testing the different generics out there, being able to document that one generic is worse than another would carry more weight than just saying you feel "worse".
or simply reporting on your experience with a new drug, say the neupro patch or the new extended release carbidopa/levodopa from impax labs when it comes out.

I think there is much value in individual experimentation of what makes us feel good, and then sharing that information. But actually designing and conducting a "clinical trial" around that just seems like playing at science.

I believe the research establishment has also been playing at science for many years; they were looking in all the wrong places and not talking to patients. I think things are better now; talking to patients is one reason science is now looking in the right places.

I think patients play a huge part in the research process. Sharing what works for us with each other and with researchers is vital. I'm not so sure about the value of a clinical trial that cannot control for all of those variables (placebo effect, absence of biomarkers, no good PD definition), patient-conducted or otherwise.

In no particular order-

When appropriate, it should be possible to enlist the patient's individual neuro as an observer by means of simple, standardized forms.

We are going to be limited in what we can attempt, especially at first, and that could make us look rather silly ("The neighbor's cat ate our rat!") It might be better to present what we have in mind as triage to identify promising and productive areas to invest the real dollars in. An example might be mucuna as an affordable alternative to ldopa.

Handled properly, the real value here might be the PR angle to force a sense of urgency onto the table. The AIDS activists were not throwing pies because they enjoyed baking. They were getting a message out. We have an easier message to convey - time's a'wastin'! The symbolism of the patient demanding a major role at the table can be a powerful one.

Some people might think I am crazy for the way I experiment, but if I hadn't gone ahead and started my first experiment of makinig fava tops tincture when I was not experiencing so many symptoms of PD....I wouldn't have been able to do it later. I was doing it for the lady that I was taking care of with PD..who was desperate for me to find something natural to help her symptoms. The fava beans worked for her , but long term storage resulted in freezer burn, etc. By the time my first plant tincture was ready to strain ...this may sound very strange to most of you....God had allowed me to start experiencing more distressing aspects of PD (one of them,, having to go to the bathroom every 15-20 min and then incontinence on top of it. Some of you will never experience this problem, but I know others have and know what I am talking about) I was ready to quit driving because my reaction time was getting so much worse (and it was not good to start with!) and other things.... This gave me courage to try a small amount of my tincture of fava plant...what did I have to loose??? I told my patient..".if iI die, tell dad I am sorry ", and took about 1/2 teaspoon of it in hot water. Go to the 4 acres to grow fava thread to read "the rest of the story"...without experimenting ON MYSELF (doing non-clinical trials) I would not be working/driving/and wearing Depends!I certainly would not be farming and still looking for things that help my PD. My brother died in Dec with PD and heart problems....my children might have PD/ and my grandchildren....there are many people outside my family who I love dearly with PD and those that have gone on. I want to do all I can to at least prevent symptoms for myself and others so we can live normal lives without drugs and their side effects. And I will continue my quest as long as God sees fit to give me breath. CRAZY..yes, but I feel good. And, I am not paying a doctor big money to experiment on me. So far I have not killed myself (and if I do, it won't be on purpose ) If I do, tell Dad I sorry..........Love you all Aunt Bean

Aunt Bean, I appreciate your tireless efforts. I wish I could gather all your posts together into one book. Keep growing those beans!

I'm not quite sure what folks mean by "amateur" "clinical" and/or "trials" in this thread but thought I'd over a few organizing principals that I use.

There are at least three buckets mentioned throughout this thread:

• Experimentation with/optimizing "treatments" (evidence based medicine)
• Research to find new treatments (interventional research)
• Learning from patients (observational research) to better understand risk factors, disease progression, disease profile etc

I think our current/traditional ways of doing all three of these efforts has limitations. Doing anything "amateurish" also has limitations.

We seek to improve any/all such undertakings by making sure the questions are relevant, the studies are created in a way in which we have a shot at getting a definitive outcome, subjects are informed and treated ethically, studies are conducted with safety top of mind, studies offer an appropriate risk/return for the patient (I'm sure there are other important considerations).

We believe in some fundamental things..

*interventional trials need to double-blind, placebo controlled to give us the best understanding of impact.

*there are probably better ways to collect more relevant, informative data from patients (in observational studies and in interventional studies) and we need to try new approaches to get that data in the mix. (I will start a new thread highlighting a "research challenge" MJFF launched this week).

*properly powering any analysis is essential or we cannot draw meaningful conclusions. But, some questions lend themselves to more tolerated variability in the responses ... the law of large numbers can be your friend when asking / answering some questions. (This is one of the reasons we want to get thousands more patients signed up for clinical trials).

Anyway, just some thoughts to add to the mix ...

Debi

You hit the target right on the bullseye. I don't see how any statistical analyses can be completely accurate until we have a way to positively say that participants have PD.

Can anyone explain that ? Or what if your control sample had PD but was not yet symptomlaetic?

Pegleg writes:

You are right that the results cannot be "completely accurate"; they are flawed in many ways. Let me add my own one: there may be many different forms of PD so the same intervention can have different effects on different people.

But the point is not whether the results are "completely accurate", but whether they are more accurate than what we have now.

At the end of the day we make a decision to take this supplement or not, whether to do this exercise or not, whether to put ourselves in this environment or not. It is better that we do this with some information than none.

Does anyone argue that it is best not to collect data?

John

From what I have read, the Cure Parkinson's Trust proposed testing at least 10 medications already approved for other conditions (and considered safe) but thought to have benefit to PWP's in the next 18 months through a format they called Linked Clinical Trials.

The goal of the program is to rapidly identify any medicatiions which could either be immediately offered to patients "off label" in the clinic or undergo more RCTs to fully characterize their efficacy. Since these medicatiion are already approved in some other capacity, the Cure Parkinsons Trust estimates 10 to 11 years of normal drug development delay would be eliminated.

I think the proposal was made at the Gran Rapids Mi. conference in Sept of 2012.

NT forum members could consider a few options.

Some might prefer to participate in one of the trials. Others might choose to consult with their MDS and try one of the candidate drugs "off label" and report their experiences here.

Either way, I think this proposal addresses the immediate need to fill badly needed symtomatic and disease modifying needs of the PD community.

Some of the top notch researchers on this site can probably come up with the list of the drugs being considered under the proposal.

I know that I will gladly participate one way or another.