[paula_w]

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