...I started out being a little facetious as is my wont, but actually the idea has some merit. It would be great PR for Big Pharma and the cost would be low compared to marketing and a write-off as well. And while the ethics would have to be handled properly, I am sure that a good research team following PWP closely through the years could find something valuable. Maybe Pfizer or GKS should start buying up those old motel sites we once discussed.

Not that I want to defend all actions of all trial sponsors...can't and won't, but I did want to throw some additional thoughts into the mix in this thread.

If we want new and improved treatments for PD, we must recognize the truth that we are all best served by a constructive contract between the patient community and industry. I'm not talking about patient bill of rights kind of stuff (which, in general and theory I support) but rather more of a fuller appreciation for what it takes for industry to maintain, let along increase its investment in PD. Whether we like it or not, no company is obliged to develop new treatments for our indication. They will only do so if there is an opportunity for a sensible/competitive return on capital for equity holders. We didn't make these rules but they are the facts on the ground and, to my mind, it is imperative that this be front and center when we contemplate anything to do with trying to advance new things to the clinic.

Biology is hard. We don't understand enough about PD to make it a slam dunk for industry to develop new products. It's not clear that success in animal models is sufficiently predictive of success in patients. We don't have biomarkers. Trials must enroll large numbers of people and follow them over long periods of time to gather data needed to demonstrate the statistically significant improvement over current treatments. Patient recruitment for clinical studies and clinical trials is chronically low in PD--adding more costs of course. All these factors make it borderline for large companies to even get involved. Some of the world's largest pharmaceutical companies don't even have an interest or active PD program...we would all be well served to do what we can to change these circumstances. Many of these elements are out of our control. Again, biology is hard. But finding ways to increase patient participation / contribution of data is one thing we can do to improve at least part of the equation.

I wholeheartedly recommend and endorse the idea that each individual patient needs to pause and ascertain the appropriateness (in consultation with family and their healthcare team) for them to paticipate in any given clinical study. But please realize that when the patient community sees industry trial sponsors as the enemy or the establishment and assume we have competing goals leading us to systematically step back with a sense of what they owe us, it could/will lead to fewer opportunities for new, transformative treatments.

Again, I'm not a patsie for every sponsor and all their decisions but without a sensible/affordable plan/expectation for patient recruitment in any study, the study could be doomed from the start. And lots of non-starters add up to no new treatments. An anecdote: at MJFF we are working on a pretty big study (still in the planning stages) where we will need ~ 400 patients and ~ 200 age and gender matched controls to participate in an observational study for biomarkers. So, no intervention being tested, but rather trying to collect clinical and biological samples over a multi-year study to see if we can validate some biomarker candidates already in the works (ie, get to the next stage toward being able to use these markers of progression as endpoints in trials)...anyway, in planning meetings experts in top academic labs and companies think we are delirious to think we can complete this recruitment at 10 to 12 sites in 18 months...they have such low expectations of the PD community. I have to think that works against us. We happen to think at MJFF that we, as a community can do better.

Additionally, the learning that takes place in clinical trials is on both sides -- researchers and patients. For instance, the plea above for trials to better utilize subtypes in clinical trials...everyone would like to do this...interest and will is not what stops trial sponsors. It is that we have yet to validate subtypes (whether they exist and how they should be defined) sufficiently to appropriately use them in trials as inclusion/exclusion criteria. Or, if we wait for all the mysteries of placebo to be resolved before any more patients participate in trials, we will be shooting ourselves in the foot. And yes there are some interventional trials where sham surgery might still be our best trial design--maybe not ideal but the best we have. Patients need to trust that trial sponsors will do the best they can -- but trials are a web of complex trade-offs of knowns and unknowns; not just costs and benefits.

I guess my point here is that participation / involvement in clinical studies might be one of the most important things that every patient can consider and act on when it comes to contributing to finding new treatments. Participation is not for everyone and not all trials are created equally, but, even doubling our current efforts could make a difference not just in accelerating progress but in guaranteeing that studies in PD continue to make economic sense for industry. And, I believe that we want / benefit from as much industry commitment to PD that we can get. More shots on goal increase our chances.

Debi Brooks

Debi –

Thanks for posting. It is becoming clear that MJFF is not part of the PD “establishment;” that to compliment your innovative approach to research, you have an ongoing, vigorous, good faith effort to increase communication with patients. This is exactly the kind of partnership that is needed, the kind of dialogue that can end our us vs. them mentality.

Transparency is tricky. Patients have never had so much information. The interview with Dr. Bartus on the Ceregene 120 phase II trial failure recently posted on the Fox website is a good example. It was refreshing to have access to his remarkably frank answers to equally frank questions. One of the issues that reemerged was the annoying presence of the placebo effect. It’s great that this is being pursued by MJFF (I know that my own doctor is involved in the discussions), but should I worry that the conundrum of the placebo be solved before other trials go forward; should I participate in a trial that might be torpedoed by the placebo effect? Or do I have too much information?

Regarding your statement, “anyway, in planning meetings experts in top academic labs and companies think we are delirious to think we can complete this recruitment at 10 to 12 sites in 18 months...they have such low expectations of the PD community. I have to think that works against us. We happen to think at MJFF that we, as a community can do better.”

Apathy is a little understood but major impediment to clinical trial participation among PD patients. It is also a major impediment to us leading normal lives. If MJFF can decipher and solve the problem of PD apathy, then maybe they’ve solved PD. (see this recent thread on “do you have trouble making decisions?”) Not only is decision making difficult (a major executive functioning function), follow through can be down right impossible at times. I would do almost anything to solve this problem.

It seems we have more questions than answers right now; research is done to find answers; how do we protect the health of the patient as we move forward? What is acceptable risk?

Thank you for engaging in the conversation, Debi.

Just a thought--bet you could obtain participants for your observational study from this forum. I know my husband would willingly participate, and I would volunteer to be a control subject. Any way to tap the resources of this forum for the study? guess you would have to determine to which of the participating centers each of the members have access--listing the centers involved and providing contact information for each center may be a way to achieve more participation than is needed.
Noodling on a friday night....madelyn

Thank you, Debbie, for providing this forum with a broader picture of research than we usually see. I think it's obvious that many of us trust you and MJFF to serve us honestly, unencumbered by expectations or rules based on the past performance of the medical and patient communities. I especially applaud yor observation that "the learning that takes place in clinical trials is on both sides -- researchers and patients."

Like others here (even the guy named Et Al) I try to report back what I can from the studies I am in, from "depression can be a part of PD and is nothing to be ashamed of and not something you can shake off with a day at the circus" to "Strattera has helped me with executive function, but it makes me more grouchy than I care to live with" (a study that has just been published).

I'm sure I'm as apathetic as the next person, but I've been in a dozen or so studies (not all for drugs), beginning in 1999, and I'm one of the reasons we know something about the psychological processes of PD over time. Proud? You darn betcha. I have a pin that says "outstanding research volunteer," and I earned it. Yes, I have wonderful doctors, but I work with them in full partnership, and the time they take to teach me is found because of and during my research participation.

I don't mean to brag. I do mean to let my peers here understand that there is a lot of reward in jumping in to an imperfect world and helping out, even taking some risks (there were no data on taking Strattera and istradefylline at the same time) and facing one's own deterioration (those cognitive tests get appreciably harder as the years go by), while discussing with advocates like the Pipeliners some of the things I've observed that could be better (like the ethical dilemma of doctors whose patients can lose a medicine that's benefitting them by the order of the manufacturer). I also hope that MJFF can see that we don't all scorn the professionals who are trying to save us.

I hope this ramble can be understood by more articulate advocates than I and by the professionals they are in touch with.

God help us all.

Jaye

pdtrials.org
pdpipeline.org

J.

[QUOTE=Debi Brooks;476218] "If we want new and improved treatments for PD, we must recognize the truth that we are all best served by a constructive contract between the patient community and industry."

I agree with you 100%, but question whether this can ever be an equal relationship when industry has time, money, and power on its side, and patients have only their disease ravaged brains to offer.

The points you raised make is quite clear who has the upperhand in this relationship. You say patients need "a fuller appreciation for what it takes for industry to maintain, let along increase its investment in PD. No company is obliged to develop new treatments for our indication. They will only do so if there is an opportunity for a sensible/competitive return on capital for equity holders." Ok, I don't like this argument, but I know it's true. Do any of the national PD orgs tell industry they need to have any appreciation for what PWP go through? Not to mention why we might find the insistence on sham surgery more than a little troubling.

If we truly want to increase patient participation, industry is going to have to see and treat patients as human beings and not subjects. This must go beyond paying lip service to patient rights and actually incorporating them into clinical trials.

I do not see industry trial sponsors as the enemy, but I don't necessarily see them as the true partner I'd like them to be. Clinical trials are the only hope for increased profits for the drug companies and better treatments for PWP. At a minimum, we owe each other a commitment to make the system better for all stakeholders.

"We happen to think at MJFF that we, as a community can do better."

Yes we can with an equal and willing partner.

Carey asks "What is acceptable risk (to patients' health)?"

Like "fair," it is in the eye of the beholder.

sheryl

Ok, I obviously don't know how to add on to my original post, so I'll finish up here.

While I obviously have strong feelings about the clinical trial process and continue working towards improvements, I am pragmatic enough to know that it is the only game in town. Most importantly, it is also the only road to a cure. I have participated in several clinic trials and will continue to do so when appropriate. I urge everyone to seriously consider participating in the trials available to them.

Sheryl

I just want to add my two cents worth here. I am not adding anything new to the ongoing discussion, but stating a personal experience. I just attended a day long symposium on PD tailored to PWP, for the first time as a patient. I was totally taken aback by the condescending attitude of some of the clinicians/scientists/pharma representatives when responding to patients questions. The speakers are highly intelligent, successful people and their answers were good and logical/ But they need to realize that its time to treat patients as equal partners/ Patients I think, need to be more forceful and not be intimidated by science. After all, its the patients head thats in the spot light!! I also feel once mutual trust is built, patient participation will go up

All these years I was on the other side of fence, wondered why patients complain, hesitate to take part in clinical trials and appear to be in a rush all the time. Now I know I tell you, it is a lot easier to be the researcher working in a lab than being a patient waiting for the research to benefit me>

Girija

They do it for primates.[/UOTE]
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We are not that different from these guys! Its even easier for me, I am a vegetarian too!! Rick, where are these primate sanctuaries?????