Robert
You know how much I care for your welfare. You've been hanging around these forums about as long as I have.

I just don't want you to miss a lifetime opportunity, so when the doctor says you still "react well to meds," does he ever see you OFF? Do you have unexpected down times? Do you have dyskinesia or dystonia?

You already can see where I'm leading - this could be a cure - and the participants in this trial will be the first to get this therapy while the others have to wait inline. Just make sure.
Peggy

I've wondered if the mixed results from some of the gene therapy studies (GDNF, CERE-120) were because the participants were too far advanced and the brains cell are deteriorated beyond repair. Maybe the government should be allowing studies that include an earlier stage subgroup where more cells in the affected regions are still alive and functioning.

I know that I certainly would consider joining a clinical study that had a chance for slowing or curing this disease. I am constantly searching for the right "neuroprotective" trial to join. However, there aren't many ongoing studies and most have advanced progression as a criteria.

Just a thought,

Gary

Under the theory of "DO NO HARM" I think the FDA will not approve this type of trial for patients with mild or moderate PD -- at least not stage 1. The government is dead set on saving patients from themselves.

GARY, it seems the proverbial rock/hard place. if you treat less severe cases, hard to see an affect and measuring neuroprotection is so difficult in pd with the day to day, minute to minute variability in pd and no good biomarkers yet. so even if YOPD'ers marched on washington and demanded money for trials, imho without better measurement tools it might be a waste of time.

that issue was discussed after the last 2 ceregene trials but with limited funds and the reluctance of less advanced pd'ers to risk brain surgery, especially if you have a 50/50 chance of placebo, seems they have to go after more desperate, advanced pd'ers.

but all that could change, research in pd is advancing more quickly than ever before, just need that one or two breakthrough discoveries, seems much more like when, not if. god i hope so.

so seems we are on our own right now for attempting neuroprotection, azilect is being dispensed i think because of that, then you have supplements which MIGHT HELP, exercise, life style, diet.

Soccertese --

Your post is right on the money with one slight caveat. Neuroprotection, at least so far, has been hard to measure because it was so subtle or did not exist. I hope soon. therapies will be developed where the neuroprotection is self evident.

seems govt is subsidizing aids meds.
what if some really expensive existing drug provides sig. neuroprotection, will we be able to afford it?

copay on azilect is darn expensive yet this imho is not a very complicated drug.
wonder what the copay will be on rytary, the yet to be approved new controlled release L/C drug from impax lab? they expect to make A LOT of money on that drug.

and as a society, do we benefit more from putting more money into neuroprotectiion research to prevent new pd cases, less severe pd or into trying to rehabilitate advanced pd'er? shouldn't even be a question considering how much money we spend on defense.

and i worry about the limited L/C generic mfgs as much as i do about new drugs, god forbid if we have to rely on chinese and indian mfg's.

I just wanted to chime in here...this particular GDNF trial is for moderate to more advanced PD. They want a Hahn & Yoehr of at least 3. I am doubtful I meet the criteria because I do not look like I have PD when meds are stable and fluid. My problem is there seems to be very few trials for moderate PD.

Laura

Gary,

If you see a potential treatment, so long as you do not have to be de novo PD, apply anyway. The GDNF trial lists stage 3 and on meds I am not even stage 1,and I do not know what I am at off meds. Researchers really need us, so I apply anyway and let them decide.

Keep in mind too that the NIH does a lot of research. The way it works there is they set up a registry and comprehensively screen you. They make all travel arrangements and pay for hotel, airfare, and provide transportation plus a daily food stipend for you and a caregiver. The best part is you do not have to go off meds for the testing! Once you pass screening, your data goes in the registry and they match you for appropriate trials. I am amazed more people do not take advantage of this.

Thanks for the info Laura. I'm going to have to get more details from you.

Peg, Laura, et al,

Today I decided to apply for the AAV2 GDNF trial at NINDS.
Laura, did you call the contact person first? I plan to get things rolling on Monday.

It seems that I fit the criteria for acceptance pretty well except for the presence of family history of PD. If there is a genetic cause, it is definitely not one of the known mutations.

When I talked with my Neuro earlier, he classified me as H&Y stage III, even though he has not seen me in the off condition. He did say that he would gladly supply any of my clinical history the trial docs might want in support of my application.

So, we shall see!

Robert