GDNF - Back to the future?

In the early part of this decade Stephen Gill at Frenchay Hospital in Bristol conducted a small clinical trial that should have changed the face of pharmacotherapy for Parkinson's. The drug in question was GDNF (Glial Derived Neurotrophic Factor), a naturally occurring brain constituent, administered by pump into the brains of half a dozen patients.

The results were remarkable and, even accounting for journalistic hyperbole, make compelling viewing (http://www.youtube.com/watch?v=gnDHMveS9_M). Patients that could barely walk before GDNF, strode purposefully afterwards. Words like "miracle" and "cure" were bandied about. And this is not normally part of the dispassionate vocabulary of science.

So what went wrong? Why is GDNF not part of the treatment arsenal available to neurologists?

Shortly after this pilot study was completed, the manufacturers of GDNF (Amgen) pulled the plug and GDNF was no longer available for human use. The reasons for this decision have been debated long and hard over many years and for much of that time the company's position has not changed. Time went by and the Frenchay study gathered dust, an interesting research curio.

But suddenly it is back in the limelight. Amgen have relented and GDNF is being manufactured again. And Prof Gill has a new improved delivery system -- more targeted on the areas of need.

So how do we go forward? It goes without saying that six patients is too few to persuade scientists and regulators that GDNF works. A bigger trial, with more detailed outcome measures, is desperately needed. This should start in autumn 2011, if funding can be found.

So watch the video, decide for yourself, and put your money where your mouth is.

6 Replies

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  • Dear Jon,

    Many thanks for your interesting piece on GDNF.

    I must admit I was following the initial GDNF trial with excited interest and was bitterly dissapointed when Amgen pulled the plug with such a feeble reason.

    Let's face it we all have suspicions that the reason was the plug was well and truely pulled was the success of GDNF would insert a rather large dent into the multi Billion $ revenue that Parkinson's medication brings the drug companies and if those suspicions are true, whats to stop it happening again????

    I wish Steven Gill and his team the very best of luck and hope that the next GDNF trial will be seen through to a successful conclusion because I believe that GDNF could produce something exciting which will benifit all of us who have Parkinson's Disease.

    Bryn

  • Thank you, Jon, for stating the history of GDNF in such an objective way. So many people pinned their faith to it; it seemed then, and still seems today, a potentially life changing treatment. I haven't seen so much interest as there is now in the return of GDNF for a long time.

    Is this trial the same or connected to the trial being conducted in the US?

    Is there a possibility that Steven Gill's trial could be on an easier trajectory to making it to market?

    With patients putting a lot of their hopes on a successful outcome, surely it would be hard to pull the plug a second time.

    Lindy

  • Thank you. This gives me hope for my future. I only (selfishly) wish that Prof Gill could run concurrent trials both in the UK and the US.

    Jean-pdplan4life

  • hi jon, could you tell me is the drug liaterim,(i think thats the spelling) the same as GDNF and if so why,in your opinion, was it discontinued

  • Sorry jon i also meant to ask could you tell me why some drugs are able to penertrate the BBB whilst others arent and is the GDNF able to do this?

  • Hello Jon, find it incredible that your post has not been commented on for 3years. The GDNF Trial is very much back on, again in Bristol and again with Prof. Stephen Gill involved with the delivery mechanism. I know this for fact as I am on it! It is a double blind trial therefore I do not know whether I am receiving GDNF or a placebo, but I do know it is exciting and at the leading edge......and gives hope.....very important for PWP. I am happy to point anyone who wants to know more in the direction of more info.

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