Thanks for the kind words, soccertese. We don't have an obligation to respond to anyone. The only reason I am here is to provide information based on my experience and to learn from the experience of others.

slapdasch may be a newbie here, but he/she sounds well informed in general, and not new to PD. The reason I jumped in is because I think that most people know that MJFF raises and spends a lot of money on research, but many don't fully understand the extent to which MJFF plays a role in the comprehensive coordination of PD research worldwide, and how they have changed the basic nature of research. They are not just all about the money.

Thanks John for your post. I have a few comments to add.
From my point of view, research priorities are quite OK. Alpha-syn based therapies, gene therapy with neurotropic factors, cell implants and vaccines have a great potential to work especially if used in combination with one another. It would be similar to cancer treatment protocols of today, a combination of surgery, chemo, radiation and immune therapy. I can even dream of a cure for PD where, GdNF-gene therapy along with newly grafted neuronal cells would replace my dead/dysfunctional neurons and a vaccine/immune modulator would take care of alpha-syn aggregation and inflammation. These types of therapies can happen in the next 5 years. Scientific evidence is there, what we need is translation of that knowledge for patient use. IMHO, clinical trials and regulatory processes need major reforms before we see anything in the market for us.
For the last ten years, I have been following PD literature, exciting research translates into clinical trials, phase 1 open label studies go well, phase 2 double blind studies "fail" and that’s the end of a promising therapeutic. What can we learn from these “failed” trials? First question is:
Did the therapeutic tested really had no beneficial value or the scientific methods used are not adequate enough to evaluate its efficacy? Yes, I do agree that biomarkers would help here, but more is needed.
It is hard to believe that any therapeutic that works well in phase 1, would lose its potency in phase 2 trials. I believe that clinical trial designs are not optimal and need to be modified to suit some of the peculiarities of PD. For edample, We need methodology that would make placebo effect (seen in most PD clinical trials) an asset to a study. Published reports suggest that dopamine is produced (not sure which part of brain) in response to many positive triggers, and hope of getting better with a new treatment can be a great positive trigger. It makes sense to design clinical trials to take advantage of that feature rather than to continue with more sham surgeries to discount its effect. I do not understand why double blind studies and sham surgeries are favored by many PD scientists despite ten years of failed clinical trials. It is time to reevaluate this strategy. I am thankful to a few scientists and many patient advocates who believe that there are better ways of evaluation and these strategies need to be considered. For more on this, See an earlier post by soccertese Interview: Roger Barker, pd research, interesting opinions on current research)
Without novel thinking and PD-specific Clinical study designs, many upcoming therapeutics will fail and years of research is wasted. We cannot let that happen.
My next issue is FDA approval procedures. First let me say that I do t hink we need regulatory agencies like the FDA to approve and monitor the use of drugs and various medical devices. However, we all know how long It takes to get a drug approved (with all the standard procedures in place), we can only imagine the time and paper work involved in approving novel agents such as vaccines and cell therapies for PD. In that context, I very much like the model Dr. Andy Grove proposed to speed up this process. Google Andy Grove and FDA for more details. Briefly, his proposal puts patients in charge of their own treatment options and goes as follows: Once the safety of a drug or therapeutic is established (Phase 1), informed and interested patients seek that therapeutic for treatment while phase 2 trials are going on. This group of patients may not fit into the criteria for a phase 2 study (too young/old, early stages etc.,) but are willing to participate and are well-informed and aware of benefits vs risks of the treatment. this way patients would benefit as well as generate additional information for efficacy studies. One example where this model would work is current NIH gene therapy trial.
These are some of my thoughts for now………….
Thanks
girija

Girija makes many good points.

In my opinion, the placebo effect makes the interpretation of results difficult but, otherwise, it's to be valued.

The Grove protocol makes sense. Essentially, it shifts the burden of proof from one of efficacy to one of no harm and it shifts the focus from the set of all PwP to the individual. Once safety is shown, thousands of people could try the drug, monitoring their progress, stopping if symptoms worsen. This could mean that some people benefit, while others don't. That's OK: rather improve things for some, rather than none. Add to this an online reporting system and we get an incredibly powerful learning environment, that could give answers in months to many PD issues.

As I see it, the fundamental difference between the priorities of researchers and most PwP is time. I have a scientific interest in all the research but, unless it's going to be available in the next few years it misses the mark for me.

What matters to me are questions like: should I use curcumin? should I exercise more? should I try transcranial stimulation? should I move house? should I use therebos*? should I dance? what's the best way to reduce constipation? The common point of all these questions is that all of them refer to potential therapies (I make no claim that they work) that are available now.

What matters to me are answers about what I should do now. For instance, on the balance of evidence available now, should I take curcumin today?

This isn't a science v non-science argument. I take the scientific approach as a given. Rather, it is a case of making the best job with limited data.

I realize the selfishness of my position. There is another constituency involved: those who will get PD in the future. They are not interested in the merits, or otherwise, of dissolving Stalevo in orange juice. They are interested in detecting and stopping PD. So, the issue is also an ethical one. Where should I put my efforts? And where should other existing PwPs put their's? And where should humanity at large put its resources?

* therebo: an unproven, potential THERapy that could also be a placEBO. Examples: curcumin, grapefruit, nicotine patches, this forum etc..

John

Girija, to whom my thanks, raised the Grove protocol. What happened to it?

John

Two simple things that can be done:

1. Use Folding @ home.
https://folding.stanford.edu/

Help Stanford University scientists studying Alzheimer's, Huntington's, Parkinson's, and many cancers by simply running a piece of software on your computer.

The problems we are trying to solve require so many calculations, we ask people to donate their unused computer power to crunch some of the numbers.

2. Anytime you use Amazon.com please use smile.amazon.com and a small (0.5 % is donated). Obviously I selected MJFF foundation. My dishwasher wasn't cleaning dishes, noticed the bottom of the silverware basket was broken, allowing a knife to jam the bottom spinning spray wonder. Ordered a new basket online for $22.00 and a whopping 0.11c went to MJFF. Those pennies can add up. Seriously simple, just have to remember to use smile.amazon.com

Tom,
Thanks for the smile.amazon.com, never knew that. I am getting people to select MJFF.

Eric