http://abclocal.go.com/wls/story?sec...lth&id=8368060

phase3 not yet funded nor approved.
hired new ceo.

With pump infused GDNF, the participants had regular refills and it was not modifying a gene. Pump infused GDNF is the only treatment that some patinets insisted was feeling like being cured. I wonder why we don't hear statements like that about nurturin or GAD. If anyone reading this has a success story from gene therapy please post in the clinical trials forum.

thank you

Is there no one from the 900 trial participants who reads this forum?
We would be very interested in hearing any personal results. We are talking about 5 years now since phase I of thiss gene based therapy was completed.
http://www.neurologix.net/products/parkinsons.html

i think there were 7 phase1 participants, 42 phase2 so not that many patients got the genes.
here's an account of the first patient to get the genes

http://query.nytimes.com/gst/fullpag...pagewanted=all

These feel good stories do not give us enough to really gauge how effective the treatment might be.

First, "he was stuck in a chair" means he was freezing? or is he was in a wheelchair?

They do not tell us how his specific symptoms changed.

Next, they do not reveal med dosage changes. Does this treatment supercede Sinemet or is it still required but at much lowered dosages?

I have heard from Duodopa participants that it is life restoring...that is implied here but we need specifics.

I would hardly call this hype. In fact, considering that is the first non-dopaminergic therapy since levodopa in the 60's; and its novel, minimally invasive delivery; and that it requires no pump, I'd think they would be promoting it more aggressively. A bit odd that locals are reporting it so must be do to diligence of some PWP in the area. I am really more surprised it is not national news.

Ceregene is attempting to offer the best of both worlds: symptomatic relief and neurogenesis all in minimally invasive viral vector.

Incidentally, there is hot debate in neurology over neurotrophic treatments...

Laura

Maybe, given the Amgen fallout, participants are now required not to discuss while in study. Also with Amgen, were people hearing that the treatment was life altering during the trial or just after the drug was pulled?

There is an article here finding Neurturin to be on par with GDNF: A Comparison

This is really interesting...here we have Matt During, a scientific co-founder of Neurologix btw, sharing his view on neurotrophic factors on PDOnline Research. He essentially equates research in neurotrophics same as "flogging a dead horse" I just love the sarcasm...check out the line on animal studies in acute PD. :

I am late to this forum, but as many of you know, I have always been a little skeptical regarding GDNF, neurturin or any growth factor for that matter, and their therapeutic potential in advanced neurodegenerative disease. Where is this compelling evidence referred to in the initial post? You mean all those experimental animal studies in acute PD models. With a treatment that has advanced to the clinic, the clinical studies presumably should carry more weight than the preclinical studies. Moreover, Phase I studies have a very poor track record of predicting success in subsequent Phase II studies for surgical therapies of PD. The three Phase II trials (at least the ones I know of) using either GDNF or neurturin have failed. It is interesting to read investigators here post comments with the statement "data-driven" or "data based", yet there is a surprising inability to accept Phase II data and believe that GDNF and neurturin just may not have a role in the treatment of this disease. After each failed trial, discussions have largely focused on what went wrong, with an emphasis on delivery. Even now, Ceregene comments (on the Phase II trial) that they were surprised that despite robust expression locally in the putamen, there was no gene expression in the nigra, suggesting this is why the study failed to reach its primary endpoint. Of course, I dont know the answer, and clearly most of the individuals posting here, the NIH, foundations including this one, and several biotech and pharma companies are prepared to throw more and more money and time pursuing the holy grail of a neurorestorative and neuroregenerative growth factor treatment. How could so many peer review studies on neurotrophic factors in models of Parkinson's Disease, and millions of dollars spent, lead to such spectacular failures in the clinic? Surely, it just means we have done it wrong, we dont have the right dose, delivery or vector system, our surgeries dont target the right region, the placebo effect is too strong, poor patient selection etc. Or perhaps the adult, and most often aged, human PD brain just doesnt respond adequately to these factors. Where is the clinical evidence that a deficiency in trophic factor signaling is related to the disease pathophysiology, specifically in the advanced (clinical) stages of the disease? Where is the "compelling" evidence that GDNF and family members induce responses in the aged human brain equivalent to those in rodents or NHPs? Are our preclinical models reasonably predictive for dopaminergic therapies, but not for growth factors?

There comes a point when it is time to throw in the towel..where would Cephalon and Regeneron be today if they had stuck to their original mission on developing growth factors for neurodegenerative disease ...why is it the PD research community has to flog a dying horse...

Dr. During is a founder and consultant to Neurologix, Inc., a company that has an interest in the commercialization of gene therapy for Parkinson's disease.

I have to chime in here and add that one of our neuros, at a highly respected teaching facility no less (world-reknowned, we were told, and I do believe that....) totally poo-pooed us two years ago when I mentioned the GABA work being done by neurologix. He is convinced PD is about dopamine and only dopamine, and seemed very unwilling to even consider something like this. It is outside of his comfort zone and certainly not part and parcel of the dopamine bandwagon that has been carrying the lion's share of PD research for decades.

The same might be going on with trophic factors, there are those who simply are convinced that's all we need and they can't get outside of that box. Meanwhile, millions are suffering and valuable money and time and being spent.

Laura mentioned docs, researchers, etc. getting lambasted for suggesting things not the norm, and I think it's a lot more common than anyone thinks.

I would love to hear more about first-hand experience with duodopa. What I've read indicates that you don't really reduce your meds that much (defeats one of our goals), and you run a pretty good risk of infection. Anyone who knows anyone who has had it, please share!

as far as neurologix, i can only state an opinion.
this is a fairly simple, inexpensive procedure compared to DBS that i assume has to be at least nearly effective as DBS to be approved. MEDTRONICS was supplying the catheters.
In and out hopefully in one day, etc., etc. Insurance companies likely wouldn't have any problems paying for it.

i would think if the phase2 results were really that good they wouldn't have any problem finding funding from investors, drug companies or even MJFF for phase3.
Again, I think it points to how PHASE1 results for invasive procedures don't predict PHASE2 so do PHASE1 need to be blinded or changed somehow? or PHASE2 design be reevaluated?