A Message from the Parkinson's Disease
Foundation and the Parkinson's Action Network
September 8, 2005
Dear Friends and Community Members:
On Sunday, September 11, the CBS
television newsmagazine 60 Minutes is scheduled to air a program
on the controversy surrounding recent clinical trials of GDNF, a growth
factor that is widely believed to have potential for the treatment of
Parkinson’s disease. This is a media event of great importance to the
Parkinson’s community and we strongly encourage you to watch it. Check your
local listing for air times.
Although none of us have seen the segment, we do expect it to feature
representatives of the key players in the issue, including the patients who
participated in the trial and the doctors who treated them. It will also
surely direct a public spotlight on the complex process of drug development,
including the rights and roles of manufacturers, regulators and of course
the trial participants (and their families) on whom the entire process of
clinical trials depends.
At the Parkinson’s Disease Foundation and
the Parkinson’s Action Network, we have taken the position that the GDNF
trial participants who, with physician concurrence, wish to have their
access to GDNF reinstated, should be permitted to do so. Amgen’s decision
to refuse patients and their doctors was a mistake because it denies them
the opportunity to continue with a potentially useful treatment, and it
denies us all the opportunity to gather more scientific data about the
long-term effects of GDNF. Similarly, PDF and PAN have consistently called
on Amgen to provide more information to the PD community about the future of
GDNF research. We believe that this was not done in the Amgen-GDNF case and
have accordingly been critical of the company and the role it has played.
You should also be aware that a group of
Parkinson’s community leaders are using the 60 Minutes story to build
support for a national drive to raise awareness of the issue and generate
support both for the GDNF trial participants and for a commitment to serious
future research on the potential of GDNF and other growth factors to help
people with Parkinson’s.
They have organized a network of leaders in
dozens of communities and have prepared useful materials for use by local
activists, speakers and people who want to write letters to the editors of
their local newspapers. If you would like more information on this
community effort, contact Ann Wasson at
anjeac@yahoo.com.
We encourage you to watch this important
television program. If you have any reaction you would like to share
afterwards, please contact either of us at
acomstock@parkinsonsaction.org
or
relliott@pdf.org.
Best regards.
Sincerely yours,
Amy L.
Comstock Robin Elliott
Executive Director Executive Director
Parkinson's Action Network Parkinson's Disease Foundation
February 11, 2005
The Parkinson’s Disease Foundation, headquartered in New
York City, today released the following response to
Amgen's announcement that it would forgo reinstatement
of GDNF to patients who were involved in recent clinical
trials of the treatment. The PDF statement is authored
by Stanley Fahn, M.D., the Foundation’s Scientific
Director, and Robin Anthony Elliott, its Executive
Director.
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
www.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
Parkinson Action Network (PAN) - Response to Amgen's decision
CONTACT: Benjamin J. Kirby
Communications Director, 800-850-4726, ext.108
bkirby@parkinsonsaction.org
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.
February 11, 2005
Petition for reinstatement of GDNF therapy by PAN state
coordinators
PDF Requests the Amgen
Corporation to offer reinstatement of GDNF to patients
who have been participating in recent clinical Trials of GDNF as a
potential treatment for Parkinson’s disease
(02/06/05) A Resolution of the Board of Directors of the Parkinson’s
Disease Foundation Thursday, January 27, 2005
The Parkinson’s Disease Foundation (PDF) supports the view that GDNF, a
neural growth factor, 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.
In the meantime, the PDF supports the request of trial participants who wish
to continue testing GDNF with full awareness of any concerns there may be
about safety, and requests the Amgen Corporation, the manufacturer of GDNF,
to make the product available for continued testing to those trial
participants and their physicians who:
- request it;
- are prepared to sign appropriate waivers that will hold Amgen harmless
in the event of complications and adverse effects, whether known or
unforeseen, and whether they occur now or at any time in the future; and,
- promise to provide continuing data to Amgen on the patients’
experience with GDNF that will enable scientists and regulatory authorities
to monitor the safety, tolerability and efficacy of the treatment.
New York City, January 31, 2005
February 3, 2005
Parkinson's Action Network requests reinstatement of
GDNF treatment to clinical trial participants in the
following letter to Amgen Inc.
January 31, 2005
Dr. Roger Perlmutter
Executive Vice-President,
Research and Development
Amgen, Inc.
One Amgen Center Drive
Thousand Oaks, CA 91320
Dear Dr. Perlmutter:
On behalf of the advocates and Board of Directors
of the Parkinson’s Action Network (PAN), I am writing to indicate our strong
interest in the requests of GDNF clinical trial participants who wish to
continue their treatment to have that therapy reinstated. In
particular, PAN supports the claim of those trial participants who wish to
continue with the treatment and accordingly requests the Amgen Corporation,
the manufacturer of GDNF, to reinstate GDNF therapy for those patients and
their physicians who:
- request it;
- are prepared to sign appropriate waivers that will hold Amgen harmless in the
event of complications and side-effects, whether known or unforeseen, and
whether they occur now or at any time in the future;
- provide continuing data on the patients’ experience with GDNF that will enable
scientists and regulatory authorities to monitor the safety and efficacy of the
treatment.
While we recognize the myriad of concerns regarding continued access to GDNF,
we strongly urge Amgen to show its compassion for these Parkinson’s patients and
their families, and the entire Parkinson’s community, by granting this request.
I appreciate your consideration and look forward to working with you in the
future.
Sincerely,
Amy L. Comstock
Executive Director
Parkinson's Action Network
Related Issues
Dr. Hutchinson's Certification (Exhibit A) at the Suthers' Trial (part
1)
Dr. Hutchinson's Certification (Exhibit A) at the Suthers' Trial (part
2)
Trial Participants Respond
Trial Doctors Respond
Grassroots Responsds
Parkinson's Action Network Conference Calls Relating to GDNF
Todd & Sherer,
Michael J Fox Foundattion, January 4, 2005
Dr.
Greg Gerhardt, Lead Investigator, Director of the University of
Kentucky's Parkinson's Research Center, January 12, 2005
Amgen Responds
