I have contacted Tom Isaacs and a researcher with Parkinson's Movement who might be able to give us more recent reports on the status of the intranSal delivery of GDNF .

The guys may also be traveling, but we should have ax report by Monday.

Anybody got freezing and frigid weather!??
Peggy

Hi All,
I received a reply from Brendan, one of the researchers involved in the study.

Hi Girija,

Thank you for your interest in the intranasal GDNF project. I no longer work on the project; it was the main piece of my doctoral thesis in Dr. Barbara Waszczak's lab. We published the first piece of this work earlier this year, where we tested the intranasal gene therapy approach in rats using Copernicus Therapeutics' nanoparticle technology. See: http://www.nature.com/gt/journal/v21...gt201428a.html.

My part of the GDNF/PD focus of the project had a lot of success in rats, and further experiments are being done by current members of Dr. Waszczak's lab to answer questions raised by the first publication and figure out optimal dosing strategies for the nanoparticle treatments. We hope to publish this data sometime in early 2015.

Making the jump from testing the therapy in rats to humans will always be a very costly and time-consuming process. Once this next paper is out, I believe it will be much easier for Dr. Waszczak's lab (or Copernicus Therapeutics) to gain funding for further testing and eventually clinical trials. Please let me know if you have any other questions!


Best,

Brendan

Sure looks like Intranasal delivery of stem cells is also quite effective to improve PD symptoms! Dramatic improvement in Parkinson disease symptoms following intranasal delivery of stem cells to rat brains!

http://www.sciencedaily.com/releases...0208163504.htm

I was in contact yesterday with Dr. Barbara Waszczak, the principal investigator for all of these studies, and she updated me on the status of her research. It is quite interesting and definitely has a lot of potential going forward. Nevertheless, she is still has a way to go with pre-clinical research before she can attempt to move to human studies. A summary of the current status:

• They have shown intranasal delivery of the Copernicus GDNF plasmid nanoparticles transfect cells in rat brains and make enough GDNF to protect dopamine neurons.
• They have been able to mark the cells in the rat brain that make the GDNF, and interestingly, can see them everywhere in the brain, including the areas that are involved in PD.
• For her next study, she needs to do a dose-response trial in rats to see if they can get higher GDNF levels in the brain with higher doses of the nanoparticles.
• She is also planning a time-course study to see how long the GDNF expression lasts after a single dose.
• Finally, they need to conduct a primate model of PD to see if the intranasal delivery works in animals with larger brains.
• All of that must be completed before she can prepare an application for a phase I human clinical trial.
• They are currently seeking funding and investors for all stages of the research

Dr. Waszczak would very much like to get support from the PD community to help her move the research along in a timely manner. I will be speaking with her later to arrange a time when we can meet to discuss how we can be of assistance. Anyone who would like to get involved and help me with this should contact me. I know it is a little more difficult for us to help while she is still conducting animal trials. However, at some point, hopefully, she will be able to move to human research and our assistance can be crucial.

Gary

Hi Gary,

I have been following Dr. Waszczak's work for some time now. She and her team are doing very exciting research. Maybe the MJ Fox foundation will help fund this important research! Please tell her that I am one PWP who would be interested in being part of a human trial.

Dr. William Frey et al also are doing similar work in this area. It sure seems evident from at least the rat/mice trials that intranasal delivery of GDNF and/or stem cells does bypass the blood brain barrier. Dr. Frey has a number of US and foreign patents in this area.

http://informahealthcare.com/doi/abs...44.2012.657720

Thanks,
Tim

I would guess we're a couple of years away from the start of any human safety studies, if they do get to that point. Still have a few pre-clinical trials left and then the application process to begin a phase 1 could be a year. I would think that process may be handled by Copernicus. I'm also not sure what Dr. Waszczak's direct involvement would be as she is a neuropharmacologist, not an MD. I will ask her these questions, and I will also find out what MJFF is thinking. However, as I said, this process is going to take time. I wouldn't even want to guess how long it would be before a potential approval and commercialization. Of course, that's a good reason to volunteer for a trial, if it ever happens, so you can get the drug years earlier.

No doubt this is an interesting drug, but there are also many other interesting interventional drugs that are much further advanced in the research process (e.g. PD01A, PRX002, Israsdipine, Exenatide, Stem Cell, and AAV2-GDNF to name a few). We don't want to lose site, and not support, ongoing studies today that have already been successfully through the pre-clinical phase, and possibly several human phases.

Tom from the UK is still traveling, and probably will have so much work piled up that we won't hear anything for a while. It looks as if Girija and Tupelo pretty well have run down the current info on intranasal GDNF delivery.

I get frustrated with all of the red tape a trial sponsor and research team have to go through. First, there are low hoops to jump through, then high hoops, and then there are hoops of fire . . . You know what I mean. It's bound to be even more frustrating for those working directly with the clinical therapy.

I've pretty well resigned myself that anything new s going to be too late for me. Saturday, November 15 was my 64th birthday, 20 of those years I was diagnosed PD.

I'm not giving up, just getting fed up. Anybody else feel the same? (As if I have to ask)
Peggy

Hang in there Peggy,

I get fed up also at the red tape involved and some studies that get funded that don't seem to have any value. Still, I get the gut feeling that something is going to break soon!

Tim