Amgen’s Decision to Block Reinstatement of Experimental Parkinson’s
Treatment For Trial Participants Is Faulted by a Leading Parkinson’s
Advocacy Group

(02/11/05) The following statement is being released by the Parkinson’s
Disease Foundation, located at 710 West 168th Street, New York City. It
follows the announcement, earlier today, of a decision by Amgen
Incorporated, manufacturer of GDNF, an experimental neural growth factor,
to forgo the offer of reinstatement of GDNF to patients who were involved
in recent clinical trials of the treatment. The authors of the PDF
statement are Stanley Fahn, M.D., the Foundation’s Scientific Director,
and Robin Anthony Elliott, its Executive Director.

"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."
********

PDF will continue to post website updates as developments occur. To read
the PDF Board of Directors resolution, which was approved on January 27,
visit
ww w.pdf.org/news/news.cfm?type=1&s electedItem=202.
For further information, contact Christiana Evers (201-960-3351) or Robin
Elliott (212-923-4792; 212-662-1957).

February 11, 2005

PAN Response to Amgen's Decision
CONTACT: Benjamin J. Kirby
Communications Director, 800-850-4726, ext.108
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AMGEN ANNOUNCES NO CONTINUED USE FOR GDNF

Parkinson’s Action Network, Parkinson’s Community Express Disappointment
in Reckless Decision by California-based Company

(WASHINGTON, DC) – The Parkinson’s Action Network (PAN) and other leaders
in the Parkinson’s disease community expressed strong disappointment with
the decision today by Amgen, Incorporated to discontinue treatments of
glial-cell-line-derived neurotrophic factor (GDNF) for people with
Parkinson’s disease.  GDNF, a promising neural growth factor, was being
used to treat 48 patients who had committed to recent clinical trials,
and was widely hailed as holding great promise as a treatment for the
more than one million American’s with Parkinson’s disease.

PAN Board Chairman Jeffrey C. Martin said: “While there is disagreement
among the scientific community about safety questions that have arisen as
part of the trial, in light of the good results from the previous English
trials, we are disappointed that Amgen did not at least allow the
patients and their doctors who wanted to continue the therapy to do so.
Many of these patients have no other choice.”

In their decision not to allow for continued access to the drug by the 48
clinical trial participants who had shown improvement in their
Parkinson’s symptoms, Amgen chairman and chief executive officer Kevin
Sharer said that encouraging patients to continue with GDNF “deters them
from pursuing potentially helpful Parkinson’s disease therapies that are
already approved and available,” referencing deep-brain stimulation
(DBS).

“While DBS is in fact an FDA-approved promising therapy, it’s simply not
a replacement for what GDNF could have provided, nor is it an acceptable
therapy for many patients,” said PAN Executive Director Amy Comstock.
“I’d like to call on Amgen to work harder to gather better information on
the population of the Parkinson’s disease community.”

“Amgen seems to have failed the Parkinson’s disease community through
their poor handling of this clinical trial at every step, including
today’s remarkably callous decision not to reinstate this drug – after
the Parkinson’s community offered them every opportunity to continue
while being held harmless,” said Comstock. “In addition, I find it
offensive that Amgen notified interested Members of Congress of this
hugely important medical decision before even notifying the doctors or
patients of their decision.  We are deeply disappointed, and we will not
stop fighting for the patients that deserve better than this.”

The Board of Directors of the Parkinson’s Disease Foundation released a
resolution January 27, 2005 “supporting the view that GDNF… is
potentially useful for the treatment of Parkinson’s disease and urges
further investigation to explore and resolve the issues of safety and
efficacy that have been raised in clinical trials to date.”  And PAN
released a similar resolution, signed by more than fifty State and
Congressional Coordinators, the volunteer grassroots leaders for PAN,
that read, in part: “More than ever, progress in medical research relies
upon clinical trial participants and sponsors willing to take risks.
Although it is understood that all clinical trials will not result in new
therapies or treatments, it is imperative that unnecessary suffering of
the volunteers be avoided at all costs.  On behalf of the Parkinson’s
community in our states, we are hopeful that Amgen will lead the way in
empowering all clinical trial participants with continuing options for
their treatment.”

Founded in 1991, PAN is the unified education and advocacy voice of the
Parkinson’s community—more than one million Americans and their families.
 Through education and interaction with the Parkinson’s community,
scientists, lawmakers, opinion leaders, and the public, PAN leads the
fight to ease the burden and find a cure.  PAN increases awareness about
Parkinson’s disease and seeks federal support for Parkinson’s research.
For more information on the Parkinson’s Action Network, please visit
www.parkinsonsaction.org or call 1-800-850-4726.

For more information on GDNF issues
see: http://www.pdpipeline.org/focus_updates.htm

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