GDNF
PLWP's Fact Sheet
Condensed Summary
Glial cell line-derived neurotrophic factor (GDNF) is a naturally occurring protein found in the brain, believed to nourish and promote the growth, regeneration and protection of dopamine producing neurons.
Indepth Info Below
Read:
PRESS RELEASE October 3, 2005
Read:
PRESS RELEASE September 13, 2005
View:
3-Min Clip of "60 Minutes"
Read:
Transcript of Entire "60
Minutes" Segment
Read:
Roger's Testimonial
Read:
Background of GDNF
Read:
Amgen's First Email Response
with
detailed
response from Jean Burns
Contact:
Amgen (Sample Letter)
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letters to Editor:
Sample 1
Sample 2
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On-line Petition
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GDNF, Amgen, and the Rights of Parkinson’s Disease Clinical Trial Participants
Glial cell line-derived neurotrophic factor (GDNF) is a naturally occurring protein found in the brain, believed to nourish and promote the growth, regeneration and protection of dopamine producing neurons.
Amgen, Inc. owns the exclusive patents to synthetic GDNF. The most recent was issued last fall, just eight days after Amgen stopped six-month-old phase II clinical trials of GDNF in 34 mid to late stage Parkinson’s disease (PD) patients. PD is a chronic degenerative movement disorder whose symptoms appear when 70% to 80% of the brain’s dopamine-producing cells stopped functioning. Its hallmarks are tremor, rigidity, slowness, lack of motor coordination, and postural instability. Late-stage patients cannot care for themselves, and often die from complications of the disease.
The Amgen trial participants say their lives were restored while on GDNF. By stopping the trials, Amgen returned them to physical deterioration and slow death. Two trial participants from New York and eight out of ten in Kentucky filed suit against Amgen to force the company to provide them with GDNF. Judges in both cases ruled in pretrial motions that Amgen was under no legal obligation to do so even though the FDA had determined that extension of GDNF based on "compassionate use" was open to Amgen.
Amgen claims GDNF was ineffective and unsafe. Trial researchers and independent PD specialists have challenged those findings, saying that clinical improvement was significant, that PET scans of the patients’ brains showed no sign of damage from GDNF, and the trials should have continued. Further, results of a brain autopsy of a GDNF trial participant in Bristol, England, published in 2005, showed re-growth of dopamine fibers where GDNF was infused. No other therapy has reversed the course of this disease.
Delivery of GDNF in the phase I trials in Bristol and at the University of Kentucky used pulse infusion through a single port with a small catheter which forced GDNF directly into the area of the brain affected by PD. This method reduced disease symptoms in all trial participants. Significantly, and against the advice of at least one of the earlier researchers, Amgen switched to a pump with large a multiport catheter that sprayed the brain with GDNF. Some of the Amgen catheters separated from the pumps and no GDNF was delivered to the brain. Amgen also lowered the dosage of GDNF in its trials.
Amgen Director of Research Roger Perlmutter said in speech given while phase II trials were underway, that although GDNF showed positive results that could not be placebo, and it could be marketed by 2006, it would not be a "huge moneymaker." The patent Amgen applied for eight days after it halted the trials included an "encapsulated" delivery system which would be far less expensive than the present pump system, but will take years to bring to market. We suspect that the increased profitability of the encapsulated version down the road was a significant, although unspoken, factor in Amgen’s decision to stop the trials and not give the trial participants further access to GDNF.
The men and women who participated in Amgen’s trials should be treated as human beings, not as dispensable lab animals. They underwent extensive surgeries, taking great risks to provide data to Amgen. They are willing to waive all their rights to sue Amgen over any harm arising from continued use of GDNF. The FDA says Amgen can extend GDNF to trial participants on a "compassionate use" basis. But Amgen has rejected this option.
The GDNF story is complex, but it is important. We are trying to bring the issues it raises before the public. We have promoted the "60 Minutes" segment on this matter that aired Sept 11; published an open letter to Amgen CEO Kevin Sharer in the Ventura County Star (September 13); started a letter writing campaign to the media and Amgen; and issued two press releases, Parkinson’s Patient Advocates Call On Amgen To Move Forward With GDNF Clinical Trials (Sept 13) and Awaiting Appeal Hearing Plaintiff Patients In Amgen GDNF Lawsuit Call Company’s "Form Letter" "Insensitive And Inaccurate" (Oct 3).
Now we come to you, as a representative of the people, to help us get GDNF for these patients. We are asking for any and all assistance your office can give. A meeting with your office and parties involved might help change Amgen’s decision, or perhaps a briefing or a hearing on the issues in this matter is appropriate.
The GDNF controversy is the flip-side the Vioxx controversy. Is it any less wrong to allow people to suffer and die by stopping trials of a drug widely thought to be safe and effective in treating a terrible illness than it is to rush an unsafe drug to market?
Currently, companies like Amgen hold all the cards. The GDNF controversy could be the catalyst for a much needed examination of patient rights and drug company practices. But our first goal is to get GDNF for the people who risked so much, gained so much, and then had so much taken from them.