Thank you, for your candid and interesting remarks, Debi. But just this morning I was talking to a participant in Phase II Spheramine trials, which as you know did not meet its endpoints (i.e. the predicted efficacy), and I want to add some thought to ponder.

The Pipeline Project, which is a grassroots group who support a speedier approval process of drug and treatment approval, have been looking at the pros and cons of sham surgery. I have heard both sides of the theory - some doctors say it is absolutely essential; others just say "it's required for approval." I am sure our fearless leader, Perry Cohen, will have some remarks to add here.

Back to my reason for talking to the Phase II participant this morning. We have had three major trials that showed great promise in Phase I trials, and up to the time of examining results statistically, were all three showing "observable improvement" in participants in Phase II, that were surprising, even shocking in some instances, when Phase II results did not meet endpoints. I am speaking of these trials: GDNF Spheramine - and the recent gene therapy - CERE-120 - all using surgical intervention - all using sham surgery (phase II) - and all open label in phase I (the participants knew they were getting the real thing). I wa wanting to hear from those who experienced first-hand the actual good and bad of the use of sham surgery in these trials.

The Pipeliners are preparing either another article or a new presentation (or both) on the ethics used in PD surgical trials. (I invite you to read our article in full published in ASENT (Journal of the American Society Experimental Neuro Therapeutics) at our website: www.pdpipeline.org )

Abstract: Ethical Issues in Clinical Neuroscience Research:
A Patient’s Perspective

Summary
A patient-centered paradigm for clinical research and medical care is presented as a solution to the problem of declining innovation and increasing costs and development time in the pipeline for new therapies. Fundamental differences in values and motivations among scientists, clinicians, industry sponsor, and patients in neurotherapeutics provide a framework for analysis of ethical conflicts and the loss of public confidence in medical research. Parkinson advocates’ views on clinical trial participation, perceived risks and benefits, placebo controls, and sham surgery are presented. These views reflect the sense of urgency and the unique perspective that comes from living with this progressive, debilitating condition full time. A patient-centered paradigm that includes authentic voices of patients as collaborators at every stage of development will help to resolve conflicts, build trust, recruit trial participants, and accelerate new therapies. Key elements are adaptive clinical trial methods and the development of information technology for the assessment of outcomes and surveillance of safety over the life cycle of a medical product. Supported by the Parkinson’s Disease Foundation, the Parkinson Pipeline Project is a grassroots group of Parkinson’s patients whose goal is to represent an authentic voice for patients in the treatment development process. This group promotes education and communication between members of the Parkinson’s community and active stakeholders in medical research, industry, and regulatory agencies. Its members are an example of a new breed of knowledgeable consumers, armed with first-hand access to research findings and reinforced by on-line connections to like-minded peers throughout the world.

Let me continue - when talking with this person this morning and bringing my thoughts together of conversations with at least two more participants who were in the gene therapy trials (one got the real thing and one got "shammed,") I don't believe anybody thought about the emotional aspects that were involved in being a participant in such trials. For some, to find out that they had sham surgery when they thought and analyses indicated improvement in symptoms - well, it was devastating to that participant.

I will not go on here for now (but would love to discuss this further) as I sam sure others will have plenty to contribute. But I do want to make a plea for anyone reading this who participated in a Phase II trial fo either GDNF, Spheramine, or CERE-120 to contact me via Private Message and I would like to talkk with you offline or via private email. In my opinion there's enough to warrant further investigation into the ethics of using sham surgery for Parkinson's.
Peggy