GDNF moving forward???

[LindaH]

The Parkinson Pipeline Project has been tracking GDNF research for a number of years. It is currently still all pre-clinical. The info below is from company press releases, journal articles on animal studies, and company websites, and the records in our database.

Our understanding is that the upcoming NIH/NINDS sponsored phase I/II study will be testing gene therapy delivery of GDNF using an Adeno-associated virus (AAV2) as a vehicle for gene transfer. This is the same virus used by Ceregene for their neurturin trial. The lead researchers are members of the Parkinson's Disease Gene Therapy Study Group, led by Dr. Howard Federoff, Dr. Bankiewicz is also one of the investigators.

MedGenesis, a small biotech in Canada just raised 5 million for their phase 2 GDNF Trial.The company obtained the rights to Amgen's gdnf protein in 2010. The protein is a bioengineered version of naturally occurring GDNF – as was used in the Amgen GDNF trial, but this time the protein will be delivered to the brain by Convection Enhanced Delivery, which is supposed to target the delivery more accurately. This is not gene therapy. There are some hints that a small phase I trial has been completed, but we don’t have details.


Dr. Stephen Gill, in the UK, with the support of the Cure Parkinson’s trust is also working on a new delivery system “to enable this key growth factor to be delivered optimally, not only in terms of safety and efficacy, but also in a way which can be administered to patients practically and accurately.”. A “two year double blind, randomized, placebo-controlled phase 2 study of intermittent intraputamenal GDNF infusions for the treatment of Parkinson’s in 36 patients. “ is planned.
See http://www.cureparkinsons.org.uk/doc...951&id=0:36727
There appear to be some connections between the two projects.
MedGenesis Scientific advisors include Krystof Bankiewicz, Steven Gill, M.D.,Howard Federoff and Roger Kurlan.
The NIH Gene Study Group includes Dr. Federoff, Dr. Bankiewicz and Dr. Kurlan.
Confusing?? YES! Perhaps they are collaborating to determine which delivery method works best?


You can view the Parkinson’s Pipeline Project database records at:
http://apexutf.shellprompt.net/pls/a...2884072126::NO
(the website is currently down, but the DB is available)

from the Scorecard , click on the Treatment column to get an alphabetical list. All of the GDNF studies should be listed together. Again, these are preclinical.

[sim00]

Thank you Linda, very interesting link (http://apexutf.shellprompt.net/pls/a...2884072126::NO).

[jeanb]

participating in the kinetics trial is NOT required to be in future gdnf trial. but if you were in the kinetics trial it puts you in the "first pool" of applicants to be considered for the gdnf trial. no word on the start -- just that they hope this year sometime.

i will finish the kinetics trial in may , i will be one of last participants to complete it.

i am sorry for my earlier misinformation.

jean

[Jeberling]

The Michael J. Fox Foundation is supporting MedGenesis for the pre-clinical development of an optimized delivery system of GDNF via convection-enhanced delivery (CED). This new method of delivery has the potential to be an improvement over previous methods that have not, to date, proved successful in the clinic.

https://www.michaeljfox.org/foundati...ng-the-barrier

Wouw, exciting, GDNF trial finally. On the other hand ... http://brain.oxfordjournals.org/content/134/8/2302

This is less promising. Seems like GDNF will only be usefull for PD patients without misfolded a-synuclein.

EDIT: https://www.michaeljfox.org/foundati...p?grant_id=770

Anyway, even if GDNF could only help a certain percentage of PD patients, that would be great. Besides that, the clinical trial could show the new system to insert the GDNF is very accurate. This would mean that other proper meds (eg. those that stop aggregation of a-synuclein, like curcumin) could be inserted in the brain via the same mechanism. So there is always hope even if GDNF isn't going to be the miracle cure for everyone.

[crimsoncrew]

Does anyone know whether the GDNF trials have started?

Also, I don't have any legal background in intellectual property law. When Amgen's patent on GDNF expires, are other groups and companies then able to openly use GDNF in their research and products?

[lurkingforacure]

Originally Posted by crimsoncrew

Does anyone know whether the GDNF trials have started?

Also, I don't have any legal background in intellectual property law. When Amgen's patent on GDNF expires, are other groups and companies then able to openly use GDNF in their research and products?

Last I knew, patents last for 17 years....so this doesn't help us much. Plus, what seems to usually happen is just as the patent is getting ready to expire, a "new improved" version appears, with its own 17 year patent!

But generally, I think yes, after the patent expires, the formula/technology comes into the open market, that's why there are generics out there. But don't hold your breath for a generic GDNF, either....:search this thread for info about generics and what that really means: only the active ingredient has to be the same. The fillers, composition, preparation, etc. can all be vastly different...which can to vastly different responses. Additionally, there are acceptable variations between generics and the name brand, often can be as much as 20%-so that 25/100 could be that much off of the name brand. If you've ever wondered why a generic doesn't work as well for you as the name brand, this is probably why.

Didn't mean to go off on all of that, it's just that so many people have no idea how different generics can be from the name brand. Even if GDNF was able to be made generically tomorrow, because of current law, the generic could be quite different from what is being used in these trials.

[crimsoncrew]

On the GDNF patent, Perry's post early last year (Jan. 2012) indicated that the patent expires in 2012.

On pdpipeline.org's website, it looks like Amgen was already doing Phase I / II clinical trials in 1996, so 17 years could have passed already!

[Conductor71]

Originally Posted by crimsoncrew

Does anyone know whether the GDNF trials have started?

Also, I don't have any legal background in intellectual property law. When Amgen's patent on GDNF expires, are other groups and companies then able to openly use GDNF in their research and products?

Looks like Amgen still has patent rights:

http://www.medgenesis.com/news.htm#financing