[Tupelo3]

I attended the NY Clinical Trials Fair this past Saturday, hosted by MJFF. I thought I would pass along a few observations. First, I think the whole concept of MJFF having these Trial Fairs across the country is a fabulous concept and hopefully will help educate PWP on research being conducted in their nearby communities and, subsequently, increase patient participation rates. The fair was very well organized with about 40 researchers and doctors available to explain, one on one, their ongoing studies. Most of the major hospitals and MDS centers in the NYC metropolitan area were represented.
I spoke to the majority of researchers at the fair.

My second thought was that there were a proliferation of interesting Observational, Genetic and Imaging studies currently recruiting patients. I signed up for two long-term observational studies, a genetic study and an imaging study.

Unfortunately, I did not see anything new and exciting with regard to clinical studies, either for symptomatic treatments or interventional for progression control. I spoke with the research team for the transdermal nicotine patch study but don't think I'll participate.

I was surprised to see that Cornell is revisiting the CoQ10 research by beginning a new pilot study. They will be researching Ubiquinol this time. Does, anyone remember what form was used in the previous study which was stopped (Ubiquinol or Unbiqinone)? They are also beginning a new trial testing N-Acetyl Cysteine (NAC) for neuroprotection. I'm getting more qualifying information on this one.

Finally, most of the researchers had the same statements regarding recruitment of patients - It's very difficult! Many of these studies have been delayed for years because the trials don't have large enough sample sizes. Clearly, this is more the case with the clinical studies than the others. They claim that as many as 85% of all studies are either delayed or halted because of lack of patient participation. I personally don't know how to solve this problem, although I have many thoughts on it. I do know that if we are going to cut down the extensive time it currently takes to take a drug from conceptual idea to available on the shelf, we as a community, have to do something about this. We owe it to ourselves, our kids and our grandkids to participate in research so we can speed of the pace of finding better symptomatic drugs and hopefully, the holy grail, of neuroprotective and progressive slowing drugs.

I will be attending another MJFF event on Thursday, the PD Therapeutics Conference. This event is geared more toward researchers, scientists, and doctors. I'm hoping that I'll get some information on new research and studies that have not yet begun.

Gary

[johnt]

Thanks for your report.

Tupelo3 mentions the difficulty that many researchers have in finding PwP to participate in trials. In my opinion, reasons for not participating are many and varied, so there is no one answer. But the points worth looking at include:

- Run the trial as much for the convenience of the PwP as for the staff.

- To some extent the number of people wishing to enter a trial reflects people's perception of the promise of the therapy. Low uptake is in a sense a vote of no confidence; this may be unfair, especially to early life-cycle trials, but it could reflect poor communication or low relevance.

- Do as much of the testing at home as possible.

- Promise to continue access to a drug for those who want it regardless of the outcome of the trial (we are all different so some drugs can be beneficial to some but bad for others. I suspect that this was the case with GDNF);

- Find alternatives to having some patients taking a placebo. (It's not perfect, but in many situations placebo figures can be shared between trials.)

- Get more information out of each participant: high frequency testing.

- Careful trial design so as to maximise the amount of information gained.

John

[soccertese]

had this open in a tab, meaning to research it more. i've tried NAC for increasing glutathione years ago, didn't feel anything but had very mild PD symptoms. FWIW
http://medicalxpress.com/news/2013-1...erapeutic.html

[Tupelo3]

[QUOTE=soccertese;1024512]had this open in a tab, meaning to research it more. i've tried NAC for increasing glutathione years ago, didn't feel anything but had very mild PD symptoms. FWIW
QUOTE]

I don't know much about the new study yet. It's being conducted by Matt Ogg at Cornell's Weill Medical College. He is supposed to be contacting me next week to give me more information and eligibility criteria. I'll fill you in if it seems interesting.

[Tupelo3]

John, some of your points were addressed at the PD Therapeutics Conference yesterday:

1.- Run the trial as much for the convenience of the PwP as for the staff. This issue came up several times. I spent about 30 minutes with the MJFF research team speaking about it also. I think everyone is in agreement that its an important issue and are trying to come up with workable solutions. Because so much of the study is specific to a lab or clinic, its convenience is not always easy.

2. - To some extent the number of people wishing to enter a trial reflects people's perception of the promise of the therapy. Low uptake is in a sense a vote of no confidence; this may be unfair, especially to early life-cycle trials, but it could reflect poor communication or low relevance.
No doubt about it. We spoke about getting doctors more involved in the process to help explain the studies and guide their patients to appropriate trials. I am going to be discussing this more with the team at MJFF.

3. - Do as much of the testing at home as possible.
It interesting that you mention this. First, several of the researchers, as well as MJFF, are working on PD phone apps for continuous symptom measurement. The issue of home testing for studies came up and there was a debate. Some, like you, thought it was a great idea. Others felt there was too much lack of control and the chance for getting bad data (e.g. you may not know for sure who actually had the phone and produced the data). There was also the general feeling that the FDA would have serious problems with the concept for actual drug approval.

4. - Find alternatives to having some patients taking a placebo. (It's not perfect, but in many situations placebo figures can be shared between trials.)First, let me state that every clinical study presented showed huge placebo effects when tested. It remains a big problem and must be controlled for. Some of the thoughts involved better imaging techniques for determining trial success which would be immune to a placebo effect.

[johnt]

Tupelo3, thanks for your latest report.

We need to measure the placebo effect. My point was to ask whether there's any cheaper ways of doing it.

Cross over testing is one way of doing it.

Another way is to use the results from the placebo group of an earlier study. So, suppose in a trial for drug A the placebo group showed an improvement of 10%, this figure could be used to estimate the placebo effect in a trial of drug B. Before doing this we would have to be careful to ensure that both A and B were similar in regards to the placebo effect - both, for instance, carried the same hype.

The results from PDMeasure suggest that the learning effect (the improvement of test scores with practice) continues for a long time in PD. This tends to inflate the results of both the active and placebo groups. However, its magnitude could be reduced by doing more practice tests.

We need trials to measure the placebo effect.

(Would the placebo in a placebo trial be an active treatment?)

John