Parkinson Pipeline Project-treatment development database, research, FDA drug approval, PD clinical trials and sponsors, patient involvement

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On this side-by-side comparison we show the total UPDRS change over the six-month period of the trial for the cohort, compared to one measurement which is a measure of tremor by a little gadget, loaded into computer. The rate of change on the right side is promising to be a whole lot more sensitive and more objective, than the current method that we measure.

So this can be done, and it should be done. And by the way, in the big scheme of research spending in the medical field, it's not very expensive. The whole thing took about two years to develop, and give or take $2 million, to do -- to design and to run.

That's issue number one, the speed of information turns. The second one is even more interesting from my perspective, and that’s the subject of failure. The most important characteristic of failure is that it is a golden nugget, something that one should polish, put under a magnifying glass, analyzed --because we don't know what you're going to find when you take the dirt off.

Let me tell you a story. Back in 1969, a young engineer working at Intel was given the assignment of figuring out why the insulator layer -- that is an integral part of every semiconductor device made since the 60s -- every once in a while allows electric current to go through. And you would think that maybe the insulator has broken down. We looked for evidence of that but found that the insulator’s good, yet in some conditions the current went through again. We look again - insulator’s still good. Making a long story short, this engineer came around and scratching his head reported, “I think we see tunneling through the insulator.” Tunneling is one of those magic quantum mechanical words that is sort of like a fourth dimension, and the space/time curvature - it doesn't mean anything to anybody who is not in the priesthood of quantum mechanics.

[Laughter]

It is not a destructive phenomenon. It's a very special way insulators can allow electricity to pass through.

We thought the guy was on the controlled substances in the 1970s.

[Laughter]

He wasn't. It turned out that he was correct. His device turned out to be THE basis of all so-called non-volatile memories which are in computers, every cell phone, every digital camera. The memories in each of these are implemented with a variation of this device. Every MP3 player also came out of the golden nugget of tunneling through the insulator. That's what can happen when we analyze failure.

Now I'm going to run through the story of GDNF, which is a growth factor protein that in 2003 was shown by a British neurosurgeon in an open-label trial to have clinical effectiveness. This lead to a placebo controlled, randomized trial, by the manufacturer of the drug, Amgen. We all know a protein doesn't penetrate the blood-brain barrier. If we want to get it to a particular place in the brain, we can't use the vascular system, we have to mechanically get it there. The protein was delivered with a form of infusion: a cannula introduced mechanically through a hole in the skull to the appropriate region and the protein in a suspension was pumped under pressure to this region. They called this kind of flow “convection”. The original paper describes this phenomenon as shown here:

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