Hello boann:
I've just stumbled across this website and have found your post. I can personally tell you much of what has been going on since the halt of the gdnf trial. My husband is one of the 34 patients who were in Phase II.

It's very interesting that the doctors are divided between their theory of whether or not GDNF works. The reality is, from a patient viewpoint, the drug DOES work. My husband has been off the drug for two years and nearly 3 months. His regression has been minor. Prior to GDNF I had to button his clothes and cut his food. He could not walk 1 block without falling along the way, the list could go on. After GDNF he has done many things, most of which has been in the newspapers already such as replacing my kitchen cabinets and building a deck on the back of my house, yes, holding a nail and hitting it with a hammer without hitting his fingers! We've done many things since however this summer he has build stairs for me and climbed a ladder and trimmed all 30 of our 15 foot tall bushes. He is not only stable but a fully functioning human being.

The patients, as well as caregivers, advocates and some of the doctors have been fighting and trying everything possible to convince Amgen they have a good drug. My husbands PET scans prove it. I truely believe, that the halt has more to do with the delivery system than the drug itself due to the high cost of the surgery and the management of the pumps. Dr. Perlmutter has made the statement in his speech at a California college that hospitals would rather see more knee surgeries than these sort of surgeries because they can move people in and out faster.

My husband had no difficulty whatsoever with the drug or the pumps. Some patients had catheters that migrated so they were not even getting the GDNF. So essentially, we go from 34 patients in the study to, in reality, 29 or 30. If 1/2 of these patients were on placebo, then essentially only 15 people were getting GDNF for the entire time. Is 15 people over a 9 month period of time a good enough sample to judge either the safety or efficacy for a population of 1.5 million in this country alone? Not a lot of common sense in that to me. Not only that but the primates were getting 10 to 15 times the dosage of humans. You take 10 to 15 times the dosages of Tylenol and I'm sure you'll have some nasty effects. Amgen didn't even take the time to consider the "Early Withdrawal" phenomenon which, I am told, is a high probability of why the monkeys developed lesions. So who is Amgen protecting? Certainly not the patients. If they would have ever taken the time to pay attention to the patients, they may have looked at the decisions made about GDNF in a different light.