Comments on Parkinson's Med: Parkinson's Patients Plea to Amgen: Third Annivers...

As an advocate against the GDNF halt, a participan...

2008-10-02T21:22:00.000-07:00

As an advocate against the GDNF halt, a participant in the Ventura county ad,snd a consultant to Nick Nelson as he wrote the moving account "Monkeys in the Middle", I have a question about the new GDNF gene.

In the initial clinical trials, GDNF was referred to as a 'molecule', a 'protein', a
'neurotrophic factor'.

Now it is being referred to as the GDNF gene. Is this the original synthetic substance that Amgen labeled unsafe? How did it become a gene? I understand that the delivery method is gene therapy,
but am confused about there being a GDNF gene.

Can someone explain if this gene is the same substance or, if not, what the difference is between the original substance, which was synthetic, and this GDNF gene?

Thank you,
Paula Wittekind

Thank you for starting this blog on Parkinson's tr...

2008-09-15T17:56:00.000-07:00

Thank you for starting this blog on Parkinson's treatments.

This month is also the 4th anniversary of Amgen terminating the GDNF clinical trial and ordering the doctors to turn off the infusion pumps of the Phase I and Phasae II trial participants(in Sept 2004). As one of the signers of the Plea to Amgen, it is deeply disappointing and frustrating that GDNf is still sitting on Amgen's shelves while the trial participants slowly returned to their pre-treatment states and all PWP are still waiting for an effective and neuroprotective treatement.

The research using alternative GDNF delivery methods, such as gene therapy, stem cells, viruses, etc. is promising, but they are only in preclinical or early clinical trial stages. According to one account, Amgen originally believed that GDNF infusion delivery could have been available for clinical use by 2007. The company still calls it a "failed trial," but if you read the later literature and reviews on GDNF research, "inconclusive" better describes Amgen's Phase II trial results. Noted were problems with trial design, dosage and delivery methods that can explain the different results between the Phase I and Phase II trials. Most subsequent articles recognized the promise of GDNF and called for further research to resolve these issues. The trial participants knew that GDNF worked. Their experiences should have been considered along with the statistics.
For more about GDNF and Amgen, I highly recommend the book, "Monkeys in the Middle" by Nick Nelson, it's available on Amazon.

Thank you for posting this. I was one of the patie...

2008-09-14T07:48:00.000-07:00

Thank you for posting this. I was one of the patient advocates in that coalition who fought for GDNF.

Maybe AMGEN in its current financial difficulties will rethink selling the patent for its synthetic GDNF.

One can only hope.