This is strictly my opinion (and please note that I think this is a wonderful discussion), but I don’t believe we can do justice in answering the original question, “Do we have an obligation to participate in biomedical research?” unless we drop the discussion of health reform. That’s a no brainer – we definitely need major health reform, but I am not sure we need a system of social medicine or just how that should be approached. One thing for certain, it should not be rushed. We got into this mess over many years, and it will not be appropriately solved if done so hurriedly. Enough said about that. I would like to answer the original question. And I am assuming the question is addressing whether or not one should actually be a clinical trial participant, differentiating that from the many of us here who are definitely “participating” in biomedical research, but not necessarily first-hand as a clinical trial participant.

I guess since I have made what I consider the “ultimate” sacrifice,” I am “obligated” to respond. Nearly a decade ago I became the second person in the world to participate in an experimental surgical procedure for Parkinson’s. The hypothesis being that by transplanting retinal cells (from a donor eye) into the area of my brain where “normal” dopamine production was deficient, the cells would continue to produce and sprinkle dopamine and relieve me of some or all PD symptoms. This was done unilaterally (one side – opposite side of the most affected side) using a system of microcarriers upon which these retinal cells would “stick.”

The trial (Spheramine) was in Phase I – the safety phase - and was open-label, meaning the small number of participants (3 men – 3 women) knew we were getting the “real thing.” At the time of my surgery, I knew next to nothing about the clinical trial process. Since that time I have learned more than the average person through first-hand experience as a participant and having friends who were participants in a number of trials and as a charter member of the Parkinson Pipeline Project, a grassroots effort to speed up the drug and treatment approval process ( www.pdpipeline.org ). Additionally, I have been working with the Parkinson’s Disease Foundation (PDF) through the Accelerating Parkinson’s Therapies collaboration (APT program) at www.pdtrials.org ) and served on the planning committee and attended PDF’s Clinical Research Learning Institute. Other areas where I have gained knowledge and experience is through volunteer work with the Michael J. Fox Foundation, the Parkinson’s Action Network, and the Parkinson Alliance. I also have volunteered to give presentations of my clinical; trial experiences at conferences sponsored by NPF and APDA, along with several other reputable organizations.

I give you my background because of what I am about to say: In my opinion the drug approval process at present is antiquated, often handled by those with only profit in mind, is not transparent, and is NOT patient-centered. To use an over-used quote: WE NEED CHANGE . . . adding . . . BUT NOT WITHOUT THE PATIENT VIEWED AS THE SINGLE MOST IMPORTANT STAKEHOLDER!

The question of whether or not we have an “obligation” to participate in biomedical research might best have been asked as “Why do people participate in biomedical research?” In 2005, a Harris Poll was conducted by PDF for the APT program. . The poll’s results showed that only one percent of PWPs participated in clinical trials; of those PWPs surveyed who were aware of trials, only 11 percent of them had received information about trials from their doctors.

Survey results such as the 2005 Harris Poll reinforce my earlier comment that the approval process is not patient-centered, or they would be the first to be made aware of relevant trials. Another factor is the difficulty of recruiting and retaining trial participants. The aforementioned “DOR or “Declaration of Clinical Research Rights & Responsibilities for People with Parkinson’s,” was written as a result of patients not being at the center of trials. Horror stories of patients picking up the newspaper and reading that a trial in which they were participating had been halted or hearing through the grapevine that the stockholders had cancelled further work in a study are just some examples why the DOR was written. (although a work still in progress this is a draft copy: http://pdpipeline.org/advocacy/rights.htm )

We hope that advocates of finding a cure or better ways to manage Parkinson’s disease will unite in the effort to educate others in what is the only acceptable and humane way to view the trial participant – i.e. as the single most important stakeholder in the approval process. If there are no trial participants, there will be no improvement or steps made forward in eradicating Parkinson’s disease. With 80 million babyboomers hitting age 60 (the average age of PD onset), I believe that Parkinson’s will be a force to be reckoned with on an equal plane as AIDS or the H1N1 virus.

By the way, in Phase II of the trial in which I participated (double-blind, sham surgery, placebo-controlled multi-facility design), the 72 participates were measured at 12 months and told that Spheramine did not meet its end points, meaning the treatment did not show efficacy over the participants receiving sham surgery. At this point of this “game” the big pharmaceutical company pulled its sponsorship, leaving the small biotech company who created the treatment high and dry – almost to the point of bankruptcy. It was then that I realized the trial was no longer a service or humanitarian act for the Parkinson’s community, but was a “business venture” gone sour. Whether or not the study will be revived or even salient points preserved for future studies remains to be seen.

Peer reviewed results of Spheramine, also known as the STEPS trial, have not yet been published – the trial was halted July 8, 2008. But that’s another story!

Sorry this was so long.
Peggy