Parkinson’s Disease Foundation Expresses Concern at Statement on Kentucky
Court Decision Supporting Amgen’s Denial of Patient Access to GDNF

(July 12, 2005, New York, NY)
The Parkinson’s Disease Foundation (PDF) is disappointed with yesterday’s
US District Court decision to uphold Amgen’s discontinuation of access of
GDNF to eight patients who participated in a University of Kentucky
clinical trial.

Five months ago (February 2005), PDF issued a statement * faulting
Amgen’s decision to block reinstatement of GDNF, an experimental neural
growth factor, to patients who were involved in recent clinical trials of
the treatment and viewed the decision as “a mistake because it denies
patients and their doctors the opportunity to continue with a potentially
useful, yet albeit yet unproven treatment, and it denies us all the
opportunity to gather more scientific data about the long-term effects of
GDNF.” *

PDF’s position has been reinforced by the recent finding by researchers
in Bristol, UK that GDNF may cause regrowth of nerve fibers that are lost
through the progression of Parkinson’s.  This evidence was found through
an autopsy of a man who had been treated with GDNF in a pilot study of
the treatment and who had died of unrelated causes.

“The Amgen case is another illustration of how crucial it is that we
reexamine how well the current medical research model supports the
interests of industry, research, and patients,” said Robin Elliott,
Executive Director of PDF.   “We need to find better ways of balancing
the interests and responsibilities of all stakeholders while maintaining
focus on a shared goal of moving therapies forward and finding cures.  A
critical piece of this is ensuring that patients’ expectations and rights
are fully incorporated throughout the entire clinical trial process.”

“The decision in Kentucky and the similar US District Court decision in
New York are extremely disturbing for the patients involved,” said
Elliott.  “PDF hails them for their courage and thanks them – along with
members of the Parkinson Pipeline Project, a very effective grassroots
advocacy group – for their leadership in defending patients’ rights.”

*********
*PDF Statement on Amgen’s Decision to Block Reinstatement of Experimental
Parkinson’s Treatment For Trial Participants Released February 11, 2005:

“The Amgen announcement, which followed a resolution by the PDF Board of
Directors urging the company to permit patients who participated in the
company’s clinical trials the option of continued access to GDNF, is
deeply disappointing to PDF, to the Parkinson’s community, and to the
participating patients," the statement reads. "However well-intentioned
the company may have been in wrestling with this issue, we believe it has
reached the wrong decision – whether judged in terms of science, or the
desires of the people who participated in the clinical trials, or the
issues of safety."
"In terms of the science, we would argue that the reinstatement of GDNF,
if accompanied by the continuing collection of efficacy and safety data,
would enable scientists and regulatory authorities to monitor the
long-term aspects of safety and efficacy of the treatment. Furthermore,
the observation of increased fluorodopa uptake in PET scans needs to be
carefully followed over time to determine if this will eventually
translate into clinical improvement. Giving up this opportunity to learn
is, in our view, a mistake."
"In good faith, the 48 patients who participated in the clinical trials
invested their time and indeed their lives in helping to test the
efficacy and safety of GDNF. This trophic factor has long been viewed by
scientists as a potentially beneficial treatment to slow and possibly
reverse the effects of Parkinson’s disease. Since the trial was halted
last summer, many of these people, supported by organizations and
thousands of well-wishers from around the Parkinson’s community, have
pleaded with Amgen to reinstate those subjects who wish to continue with
the treatment and are willing to be carefully followed to generate
ongoing data. We understand and fully accept that such reinstatement of
treatment should take place only with appropriate waivers that would hold
the company harmless in event of any complications, whether foreseen or
unforeseen, and with the approval of the relevant Institutional Review
Boards."
"The safety issues of neutralizing antibodies (found in some of the human
subjects) and of cerebellar degeneration involving high-dosage GDNF (in
some non-human primate subjects) need to be investigated and understood.
If subjects are willing to offer themselves for continuing GDNF infusion
with all of these safety concerns fully explained and understood by the
subjects, then the Parkinson community – science as well as patients –
can gain much new information related to the safety of GDNF infusions."
"In summary, the decision is a mistake because it denies the patients and
their doctors the opportunity to continue with a potentially useful,
albeit yet unproven, treatment, and it denies us all the opportunity to
gather more scientific data about the long-term effects of GDNF. We hope
very much that this company, which has long held a position of high
respect in the business and health communities, will consider reversing
its decision, and soon. In the meantime, the coalition of
patient-voluntary groups – which besides PDF includes the Parkinson’s
Action Network and the Parkinson Pipeline Project -- will continue
fighting for patients’ interests as long as it takes."
The authors of the PDF statement are Stanley Fahn, M.D., the Foundation’s
Scientific Director, and Robin Anthony Elliott, its Executive Director.


For further information contact: Ronnie Todaro at (212) 923-4700 or via
email at [log in to unmask]

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