Oh that felt good to swear. I wonder if it will show up.

Amgen GDNF Phase II Clinical Trial - still moving on it.

Many gave up before we started two years ago. Those people had various reasons. They believed in what Amgen found - they had their pick of which to believe - Amgen had all the areas covered. They halted because:

it didn't work
it wasn't safe
the delivery system

This was a screwed up trial and the results were inconclusive.

We've been like obsessed investigative reporters ever since, because we happen to know that it works for some people and that it works WELL. That makes it no different than any other treatment, except for one thing - it regenerates cells.

We have met with the researchers who followed Amgen's instructions to remove the pump/catheters .....in other words surgery.They did what they were told. But some of the doctors felt that this was so wrong and that they were so incorrect, that they didn't remove the pumps. Two years later, some patients still have the pumps/catheters in and receive saline - waiting for it to be given back.

This was a phase II trial. It was a lousy comparison to Phase I, which wasn't blinded and all 15 people improved. Placebo they say.

It's harder to find something that isn't different from phase I in Amgen's Phase II, which had the purpose of comparing to phase I and looking at safety and efficacy.

Amgen's GDNFscientist died of a heart attack before phase II or during = i'm not sure. This is important because he was its champion and his replacement didn't fight for it. We wonder if anyone was knowledgeable enough to judge whether it should have been halted or not.

The Amgen trial was conducted with a different pump, a lower dose, and the participants were not matched. No big changes or anything. Right.

But in spite of the trial flaws, some people improved dramatically....the phase I participants were still on it all the time up to the halt. One of them in England, was on it for 2.5 years, had it snatched away abruptly and with no warning 2 years ago when it was halted, has had pd for 31 years and is STILL NOT BACK ON SINEMET. This is a regenerative drug and it works for some people- even at a lower dose than the first trial.

Although the safety factors included antibodies, which went away in some [we haven't talked to them all so can only at best say "some"] patients when the drug was halted; dislodged pumps....some if not all of which were the same people who developed antibodies because the pump "migrated" causing a leak. Third, they found lesions in a few monkeys - I think four. THese monkeys were on very high doses. and then it was abruptly halted and researchers, even within Amgen we have heard, believe that is likely the cause of the lesions. People on this drug not only don't have lesions, they don't have side effects at all except antibodies that have thus far either gone away or caused no harm.

In other words, you have a solid group of patients who say GDNF is like a "CURE" altho it isn't really.

Here's what I want to impress upon you as a pd community. If you get cancer, you might have to take chemo and radiation to get better. The treatment can also kill you. Cancer patients have that choice.

We don't. We suffer longer than cancer patients and die of our illness. This is not a competition over who has the worst disease but it's time to correct a few misperceptions. There is much pain, and much more social isolation and humiliation. The last few years we will lay there frozen and praying for death.

We need this pump infusion therapy and we need to treat people NOW. Throw in anything new. and you have to start at phase I - it takes 14 years to go thru the pipeline, FDA, and become a treatment...for the rich, until you get it covered by insurance.

We need to be given the chance to take the same risks as cancer patients. We need to have special considerations due to it being a terminal illness. Waiting for gene therapy or cell delivery is not likely for people in their 50's who are getting advanced.

The FDA approved compassionate use for those who still had equipment in - Amgen said no. Two trials for compassionate use - not money = Amgen won. We met with pro Amgen researchers for 3 hours, around a rather small table, eye to eye. They said the drug wasn't safe, didn't work. They had removed their patients' equipment. Many got DBS instead.

The FOX FOundation started out with faith in Amgen, taking them at their word. NOw they too view it as being an inconclusive study. We were invited to meet with the Fox staff and tell our story. They are good people, but Amgen remains unmoved.

Now we are giving it one last push - a big one - we are trying to get a meeting with Amgen - with the people directly involved. One of the higher up decision makers has said he will consider it and says he is getting death threats.

I told Peg to stop doing that...LMAO....JUST KIDDING. From what I've read, there are several issues for which Amgen could be getting death threats.

We just might get this meeting. Amgen exec is picturing a group of emotional patients attacking him. He's in for a surprise, hopefully not an unpleasant one.....if he doesn't review his stuff, we will probably know the study better than he does.

We are not out to hurt anyone's reputation. Removed the rest of this thought. We've been gracious.


Other than that there is only spheramine...peg can update that..but they are moving slowly.

You may not really think there wouldn't be something to help you in your lifetime. Perhaps you are early onset and it isn't bothering you; doctors are telling you they are working on it; it;s the illness to have, it's all Bush's fault.

But you don't get to start at the beginning and find this out the long hard way, you should be fighting with us now. It affects you too - bio techs determine whether you live or die sometimes. And this is one of them

Let us take the risk.
paula

We should buy all the stock, of Amgen, so in turn we could
own the GDNF Formula...

Yes! my little note to the bastards at Amgen...
erhmmm -
Kiss my shakey ****! *haha!

As usual Paula, you are right on the money..I hadnt thought about comparing the risks..(if there are any)..of GDNF to chemotherapy and radiation..We know that chemo and radiation are nasty, and destroys the body along with the disease, and makes patients sick as a dog..There isnt any sickness or destruction of the body that we know of with GDNF thus far..Problem is..chemo and radiation are profitable, and GDNF is not, and the ratio of cancer patients to pd patients is much much higher, which IMHO means that once the million and a half pd patients are well on their way to recovery..there will be less patients in need of GDNF and pd meds..(less profit)..whereas there will still be millions more cancer patients in need of treatment simply because there are more of them compared to the number of pd patients..I guess what Im trying to say here is that theres more money in treating cancer patients with chemo and radiation because of the very nature of the treatment of cancer vs treating pd patients with GDNF and that is the ONLY reason why Amgen pulled the plug..It will cost them money in the long run to treat us because of the lost revenue in the need for pd drugs..When I met Steve Kaufman at the WPC he was the picture that spoke a thousand words..He was a GDNF Clinical Trail participant and you would NEVER know by looking at him that he was confined to a wheelchair before receiving GDNF therapy..And at that time I met him he hadnt received any GDNF in 17 months, and he could walk as good or better than I could and I had had pd for about 3 1/2 years at the time..Amgen can say what they want, but I know what I saw, and Amgen is full of **** period!!..GDNF works, and it is gravely unfortunate that the research was discontinued because it held some much hope and promise..This was the answer to Bush's "problem" with stem cell research, not that Im implying here that stem cell research should not move foward..The pro-lifers would have no reason to object to GDNF therapy, and maybe Im all wet here, but maybe the concept should be sold the pro-lifers as a way of getting GDNF on the radar screen untill we can get stem cell legislation passed, then we would have the best of both worlds..I dont know if this angle would change anything..Just a thought

HGH (human growth hormone), discovered and chemically characterized and synthesized decades ago. But that's where we learned that peptide (chains of amino acids) are just too fragile to make it to the areas in the brain where they are (or are not) produced naturally.
My guess is that there just wasn't anyone with the expertice at Amgen to REALLY address this decades old question of delivery. Remember what new pharmacologists are taught; "ADME" Absorption, Delivery, Metabolism, Excretion. If only we could find a genuinely workable method to deliver GDNF to exactly where it's needed, then we could better study it to see it's real efects and promise as a PD therapy. But...............

cs,

The pumps worked for many of the patients with no side effects. We hope to be able to determine if this was a business decision or just being clueless to what they had. A recently published study found it didn't disburse far enough, but something's gotta give with experimental drugs and situations like this. How do you justify letting people suffer. Earlier statements from Amgen said, they knew it wouldn't be a moneymaker but they were going for it anyway because they could see in the imaging that it worked. It was the lesions that scared them in the monkeys, but no humans have had any side effects. I think the scientists death left it with no champion.

Welcome back and hope you stick around.
Paula

problem with the work they are doing with Ceregene.

What is the relationship between Ceregene and GDNF. I understand Ceregene are not working in the GDNF space rather with another related neurotrophic protein, however if the Ceregene trial "works", what are the implications for GDNF delivery ?

Aftermathman.

Hi aftermathman,

Ceregene is gene delivery of Neruturin, which is a member of the GDNF family. Fox is planning a gene therapy trial with GDNF I think...but better check on their site.

Amgen's still got the GDNF, but we still haven't given up. Did you know the BCC radio oro is it tv is letting Tom Isaacs do a documentary over there on GDNF?

Also interesting things llike the un of minnesota is teaching a Grand Rounds on '"GDNF ETHICS" at neurologists request.

We are attempting to meet with Amgen...it looks farly promising thanks for asking and welcome back.

Paula

the videos you posted on the old site re. the trial participants was amazing.

You may consider posting them again on this site for any new members.

Good to speak to you again,
Aftermathman.

apparently amgen is doing an observational study - they are calling in Phase IV (which means i don't think i understand the true nature of Phases of studies) on the, or some of the, participants in the two GDNF trials:

http://www.clinicaltrials.gov/ct/sho...148369?order=1

old news, right?

thanks,
boann

I'm kinda sitting in my tree up here in Canada and trying to follow this story as best I can. But there are a few things I'm not clear on. Did allthe patients in the GDNF trials (with the exception of the Amgen one that's inconclusive) benefit from GDNF? In other words, did it work for everyone who recieved it in the Bristol study, or just a percentage, and if so, does anyone know what the precentage is? I'm asking about the Bristol study because it seems to be the longest, the best run and was not double blind.

I am unclear on which Dr.'s think GDNF has value as a viable treatment and which Dr's are holding the Amgen line. I was under the impression some of the Dr's at Amgen were not happy with the way the corporation stepped in and stopped things. That there are Dr's among the Amgen research team who feel the trial was "Questionable" (picking my words carefully). Is that not correct?

How many Dr's/researchers (people who would be publishing or overseeing these projects) are estimated to be out there...10, 50, 100 whatever?

The courts have to uphold the letter of the law and Amgen clearly has that covered. Isn't the best chance for GDNF research to be kept alive from where it left off is if these Dr.s speak up (regardless of the ramifications to their careers) and support those they took an oath to serve? I don't envy any Dr. in that position if that's the case. Do I have it right?

Lastly, it's sure a sorry statement that it takes a death threat to get one human being to help another human being. Are we surprised?