Please read the entire post before replying...thanks

Hope I am not throwing myself to the wolves

I have been researching this surgery and have found very little that is available to a non-paying individuals. There is tons in the medical and science journals arguing the pros and cons, but they are only available for a "nice" price, and the abstract is usually two or three useless sentences or non-existant.

If you have any citations that you can provide on the pros and cons of placebo/sham surgery to the brain, please provide them.

I am also curious...and you Pipeliners put down your hands ...just who in the forum is completely educated on this topic. I know many are against it, but can you cite facts as to why you are against it. Yes, I know about risk of infection, allergic reaction to anesthesia and the long list of usual surgical side effects.

It would also be nice to hear from anyone who is for this type of trial, not just those against.

With regard to CERE-120, the physicians/surgeons I have spoken with state that this particular trial can not move forward without the double-blind factor. That it is imperative to measure the placebo effect and this can't be done without the double-blind factor. And, please get past the "they just want to make money and they just want to get paid for being trial investigators." I can't believe that every trial investigator is that greedy.

There are several online references to the sham surgery trials of the late 1990s. In those the placebo effect was quite evident.

Regarding any therapy that comes down the pipeline and requires placebo/sham surgery to the brain (or any other part of the body), now or the future, if the medical and pharma communities refuse to debate the issue with the patient community and believe that an acceptable outcome can not be found without placebo/sham surgery, what then will happen to therapies like CERE-120...the most promising to come through the pipeline (since GDNF)...if patients do not volunteer for the trial?

Do we simply let the "promise" of a better future for the already diagnosed, and those yet to be diagnosed, just go away...get shelved because no one would "test" the product?

Because the consent form issues are not yet resolved between them and us, and the Patient Bill of Rights is not in place, do we go on strike until it is resolved?

Of course, I agree that each and every PWP and the people who love them should be behind improvements to the consent form.

We should all be behind the Pipeliner's efforts to promote the Patient Bill of Rights. (You have no idea how many months of sweat went into preparing that document! No one kept track. In a PWP-effect I suppose the hours are immeasurable.)

We should all adopt the bumper sticker: "If you're not angry, you are not paying attention!"

No I am NOT being naive...I am questioning...as anyone SHOULD DO!!

I completely agree that you should not be aware of which drug you receive in order for the results to be valuable and unbiased. I beleive the contract for this particular treatment should include protection for the patient which should be the main ethic of the researchers. Therefore, I suggest that the contract should include a clause offering to remove the device and/or if the device causes damage to the patient some of the damage should be repaired by the researches either by surgically repair of the damage, if possible, or to compensate the family if a fatality occurs. It is only fair that if the Industry is going to profit from the risks of the patients participation, the Industry should take responsibility for any harm caused by their research of patient volunteers. The patient will then have assurance that his safety is of primary importance not just the result of the study. This assurance will cause the Industry to take on costs of the risks. I also believe the patient is entitled to all the results of the study, whether it is successful or a failure to make decisions on what further research they become involved in. All of this should fall under the category of research overhead for the pharmaceutical companies, and money put out should be taken out of profits and not passed on to the consumer as higher cost of therapies.

Vicky

P

I understand your point but u are bucking an acceptable way to get answers.
there are 10,000's of us behind u waiting to hear whether this gene therapy is a partial way home. Most of us are in early and moderate and are precluded from the sample. I would venture to guess is that if they opened the study up to us they would have no problem filling in the dance card.

Your concern is obvious and justified but if I were in the sample the question I would be asking is at what stage in the after surgery observation will I know if the gene therpy is working sufficient enough to open the study up for compassionate reasons.

good luck
gems

Gems,

Having a name that begins with a p, I'm curious about who you are addressing the reply to.

Willingness to undergo sham surgery is as individual as anything else. I'm not sure how you completely prepare for it. If you are in a trial and on the placebo, and the drug company decides for any reason to cancel the trial, you've just undergone major surgery for nothing.

There should be a safety net for both the physical and psychological consequences that may result from that.

Also, sham surgery requires more anesthesia to keep people from knowing who got the real stuff and who didn't.

IF it is me you are addressing, then I would sum it up by saying that the current system of conducting clinical trials isn't working well enough or quickly enough. The GDNF cancellation was recognized around the globe as a trial that educated and woke people up in our community. It showed us that the sponsoring company can be imcompetent about some things, that they can be arrogant and appear to lack compassion. But mostly, inspite of FDA approval to continue compassionate use with the participants, it showed us the patients are not the top priority. That's not a safe place to be when you have equipment in your body and no drug to run through it.

I wish you could help us too. One of the best things a pwp can do to help himself or help others is to participate in a clinical trial. But before you get to see if it works, you have to go through the surgery. We are looking at the ways they are trying to get around this.

Hope that addressed your comment and you probably weren't even talking to me.
paula

edited to add this link - way over my head in price and content but just sharing it to let you know they are working on these things.

http://www.exlpharma.com/eventAgenda.php?id=77