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.
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:
(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.