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Originally Posted by Tupelo3
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
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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