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PD Organizations Respond

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 Parkinsonís disease.  This is a media event of great importance to the Parkinsonís 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 Parkinsonís Disease Foundation and the Parkinsonís 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.  Amgenís 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 Parkinsonís 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 Parkinsonís. 

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 .

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 or  

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 Parkinsonís 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 Foundationís Scientific Director, and Robin Anthony Elliott, its Executive Director.

Amgenís Decision to Block Reinstatement of Experimental Parkinsonís Treatment For Trial Participants Is Faulted by a Leading Parkinsonís Advocacy Group

(02/11/05) The following statement is being released by the Parkinsonís 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 Foundationís 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 companyís clinical trials the option of continued access to GDNF, is deeply disappointing to PDF, to the Parkinsonís 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 Ė 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 Parkinsonís disease. Since the trial was halted last summer, many of these people, supported by organizations and thousands of well-wishers from around the Parkinsonís 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 Ė science as well as patients Ė 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 Ė which besides PDF includes the Parkinsonís Action Network and the Parkinson Pipeline Project -- will continue fighting for patientsí 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


Parkinsonís Action Network, Parkinsonís Community Express Disappointment in Reckless Decision by California-based Company

(WASHINGTON, DC) Ė The Parkinsonís Action Network (PAN) and other leaders in the Parkinsonís 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 Parkinsonís 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 Americanís with Parkinsonís disease.    

PAN Board Chairman Jeffrey C. Martin said: ďWhile 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.Ē 

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

ďWhile DBS is in fact an FDA-approved promising therapy, itís simply not a replacement for what GDNF could have provided, nor is it an acceptable therapy for many patients,Ē said PAN Executive Director Amy Comstock.  ďIíd like to call on Amgen to work harder to gather better information on the population of the Parkinsonís disease community.Ē 

ďAmgen seems to have failed the Parkinsonís disease community through their poor handling of this clinical trial at every step, including todayís remarkably callous decision not to reinstate this drug Ė after the Parkinsonís community offered them every opportunity to continue while being held harmless,Ē said Comstock. ďIn 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.Ē

The Board of Directors of the Parkinsonís Disease Foundation released a resolution January 27, 2005 ďsupporting the view that GDNFÖ is potentially useful for the treatment of Parkinsonís disease and urges further investigation to explore and resolve the issues of safety and efficacy that have been raised in clinical trials to date.Ē  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: ďMore 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 Parkinsonís 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.Ē

Founded in 1991, PAN is the unified education and advocacy voice of the Parkinsonís communityómore than one million Americans and their families.  Through education and interaction with the Parkinsonís community, scientists, lawmakers, opinion leaders, and the public, PAN leads the fight to ease the burden and find a cure.  PAN increases awareness about Parkinsonís disease and seeks federal support for Parkinsonís research.  For more information on the Parkinsonís 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


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