This is taken from a post I made on another PD forum:

A couple of years ago, I felt so sorry for the Amgen PD trial participants when many who had been helped - not harmed - by it demanded the right to continue to receive the treatment. And that's where I am now - dreading the end of the study I'm in next month.

The KW-6002 (Istradefylline) drug study began as a double blind event for me. And, from the very first dose, I felt so much better that it was obvious to everyone that I was getting the real drug - not a placebo. The experimental drug has helped me so much. When the qualifications for being in the study changed and I no longer qualifed to be part of it, my daily functioning declined considerably. The dramatic change in my well being on vs. off the drug led me to the decision to have surgery so as to once again qualify for the study. Yes, this drug helped me so much that I had surgery so that I could once again be part of the study!

Nearly two years (or is it three?) of taking this med and I have had no negative reactions and a noticeable imiprovement in my daily functioning. Despite this drug helping me and a lot of other PD patients that I've spoken with, the study will end and the drug will no longer be available to me - to us - as of next month.

QUOTE:"The option for participants in the experimental/treatment group to continue the treatment. Likewise, those who received placebo or low dose treatments, or sham surgery should have the option of receiving the experimental treatment at the full dose, upon conclusion of their trial."

If I understand the quote above taken from the proposed Trial Pts Bill of Rts., this bit means that drug study patients could opt to continue to take the study drug after the study ends and the study's sponsor would have to continue to produce the drug in quantities large enough to meet trial participant needs. I'm all for that!

I would sign whatever waiver they came up with to protect the drug company doing the study, but I should have the choice to continue the drug or not. This choice is not mine - I have no say in it. I think the purpose of the Trial Patient Bill of Rights is very necessary.

Considering this experience, will I participate in other drug studies that may arise? Maybe. Maybe not.

The end of this drug study is not only my personal loss, but considering the difference it made in my well-being, I think it's a huge loss for the PD community as a whole.

"Monkey in the Middle" seems a very appropriate name for a book about the Amgen trials.
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Hi DJM,

Are they still working to comply and get it approved? We are looking for the latest information. Jaye, if you are reading maybe you can add something - Jaye is in the Johns Hopkins study with it.

By now, I realize that we just don't look sick enough on the outside far too long for people to understand just how sick we are. We quit our jobs but don't get approved for social security. I'm not sure you can measure it exactly. You may know that you feel better, but it doesn't necessarily show in the doctor's office. And of course, they don't take the participants word for it - but call it placebo.

This is the link I have on it -
http://www.kyowa.co.jp/eng/netext/er080228_01.htm

DJM, my heart goes out to you. I hope there is still a chance for it and it doesn't get stuck in the FDA black hole for too long. There is a push for legislation for better access; as they say on the fastercures.org site - "something's got to give."

These are humans, not animals. Humans know what they are being denied and why. It absolutely stinks.

paula

I tolerated Istradefylline well. I took it at 0600 and my best time was in the mornings. Apparently it is different than agonists in that it makes your brain think it is producing it's own dopamine. I would definitely be taking it now if it was available.

I continue reading Howard Brody's book"Hooked: Ethics, the Medical Profession, and the Pharmaceutical Industry" (Rowan & Littlefield Publishers, Inc, 2007) Dr. Brody has an MD and a PhD in Philosophy, evident in his writing, ie dense stuff at times.
He notes the passage of the Byah-Dole law in 1980. This law was passed based upon a study in the 1970's, showing that of the 2800 patents held by the US govt and its scientists, only 5% had been licensed to manufacturers. The inference was that these discoveries needed to be moved from the laboratories into industry in a more "streamlined" way (p.75). With passage of this law, universities and the NIH could patent scientific discoveries and grant exclusive manufacturing to pharmaceutical firms to produce the final products. The law included a clause for "reasonable pricing". If a pharmaceutical co developed a product from that which was developed and patented by the federal government, the price of the product must be "reasonable" or the government had the right to repossess the exclusive patent rights from the company. This was termed the "march-in rights".
Brody notes that there was not much use of the Bayh-Dole law until 1995, when the NIH "dropped the reasonable-pricing requirement altogether" (p78). In 2002, 2 Washington Post reporters, Peter Arno and Michael Davis, wrote an article noting that the Bayh-Dole provision should give govt a way to make drug pricing reasonable. Senator Bayh reported at this time that his interpretation of the "march-in-rights" was not to affect drug pricing but to force drug cos who had been given exclusive rights to manufacture a "useful" drug and then failed to bring the drug into production to give the "public access to the new drug---at whatever price the company decided to charge" (p. 79)
I assume in all the legal back and forth with Amgen, the use of the "march-in-rights" was considered....just noodling here...The author notes that the NIH dropped the "reasonable pricing" provision--he does not state that it was repealed.....Perhaps an inquiry to either or both of the Washington Post reorters might be in order. madelyn

The theory that PD is a disease of many different etiologies, the given that some therapies may help one subset of PD patients but not others, would cry out for "compassionate use" of clinical trial meds for that group of patients for whom the drug is effective.
madelyn

Hi Madelyn,

'march in rights' were discussed, thanks for explaining it. I don't know the details about why it wasn't invoked. That might have been on the patient site that is now offline. If i find anything I'll post.

Regarding compassionate use - the FDA left it up to Amgen. FDA gave them the OK for compassionate use. It was Amgen's call.

paula

Perhaps "compassionate use" should be part of the study protocol? and for those invoking disincentive to pharma to develop new drugs, esp for diseases that occur in less #'s than would make it profitable--remember the "orphan status" drug laws (tax break and 7 yr monopoly on drug sales). and the incredible fact reported by Dr. Marcia Angell (The Truth About The Drug Companies: How They Deceive Us and What To Do About It. New York:Random House, 2004.)that in 2002, a listing of the combined profits of the 10 pharmaceutical companies in the Fortune 500 --$35.9 Billion-- was GREATER than the combined profits of the remaining 490 companies--$33.7 Billion. Dr. Brody lists the above facts in these terms: "...the pharmaceutical industry accounted for 2 percent of the Fortune 500 Companies, but 52 percent of these companies' total profits ." (H. Brody, "Hooked", New York, Denver: Rowan & Littlefield Publishers, Inc. 2007, p. 57)
Granted there were only 10 pharmaceutical cos in the fortune 500 listing, still seems egregious to me.....actually more so.

(i apologize for lack of underlining--I do not have that feature on my tool bar and have no idea how to access it.)

Sorry to intrude on the jubilation, but realistically, Americans no longer trust the government, the pharmaceutical companies and the media. The author of the book is telling the truth. So lets (Parkinson Advocates and some patients) gang up on the pharmacy (Amgen) by making them the evil one. But so many different folks with an agenda to peddle have used the "Real Story" TV programs to get their agenda "across to the public" that these programs no longer have an effect.

The ethicist has a point. We don't know the whole story, but here is an educated guess. Parkinson's is NOT a terminal disease. GDNF, given in higher doses, killed animals. The dosage to the monkeys was larger, therefore the longterm effects came quicker. The patients did have slowed improvement. The monkeys died of brain lesions could have died by overdose, but more likely the effect of the drug, as their symptoms were also noticably improved quicker than the human participants in the study. This being the case, the patients are telling the doctors to allow them to continue a drug that proves to kill (improved life quality but certain death) rather than live with a non-terminal illness and retain the possibility of a future. As a practicing doctor who promises to "do no harm", would you feel your rights as a patient surpasses a doctors' upholding his medical oath? The oath is for the patient's benifit. One exception, and soon another. All mandated by the Food and Drug Administration or National Institute of Health. Where do we draw the line and when do we start trusting again? What has happened to our Nation?

Vicky

Vicky,

There is a lack of compassion factor at work here that is a bigger player than most would suspect. One participant even said, "if they would just have told us; showed some respect. They were so cold."

People who are ill need compassion and that isn't a priority with bottom line people; it can't be. A major conflict. They don't even feel a need to explain, or at least they didn't then.

As for the nation; that would take a book.

two cents,
paula

I love Sticher's quote by Helen Keller in her signature:

"I long to accomplish a great and noble tasks, but it is my chief duty to accomplish humble tasks as though they were great and noble. The world is moved along, not only by the mighty shoves of its heroes, but also by the aggregate of the tiny pushes of each honest worker. "~~Helen Keller

Do you suppose it is great because of the person who spoke them or because of the meaning?

We must accept our limitations and respect and trust those who put their training and economic status on the line for our benefit. No amount of compassion will make your illness improve or cure it. Ethics is not about emotions. Ethics is about appropriate behavior. I love Helen Keller's quote because it is ethical not because Helen Keller was deaf and blind. That is what makes her a hero.

Vicky