The Pipeline Goes Mainstream

 National PD Organizations Initiate Collaboration With Grassroots Advocates

Aug/Sept. 2003
By * The Pipeline Group

On the ninth floor of a plush law office in Washington, D.C., a strategy toward accelerating the cure for Parkinson’s disease is being discussed. Gathered around the conference room table are representatives from the FDA, the National Institute of Neurological Disorders and Stroke (NINDS), the Michael J. Fox Foundation, the Parkinson Alliance, the Parkinson’s Action Network, the Kinetics Foundation, and the Parkinson’s Disease Foundation. Leading the discussions are Robin Elliott, PDF Executive Director and five people with Parkinson’s who call themselves “Pipeliners.” They have come from California, New York, Tennessee, and Washington to be heard – and this time someone is listening.

The slide presentation begins, and all ears absorb what this group of volunteers has to say about the Parkinson Pipeline Project (PPP). “FDA approval takes an average of 14 years from discovery to the drug store,” begins Perry Cohen, national PD advocate and the creator of the PPP. “We can show you how the evaluation and approval of new treatments and drugs for Parkinson’s disease can be accelerated through this project.” Bill Franklin, a former highly successful business planning manager from California adds, “Fourteen years is unacceptable; we can’t wait that long.” Bill is applying his expertise toward this initiative because he believes that with a talented and dedicated team the PPP will evolve and make a major contribution toward curing the disease. Representing hundreds of thousands of people with Parkinson’s this small group of people put this effort together online and made this trip to team up with the mainstream PD organizations. And no one wants to find a cure more than this group, having all been diagnosed with Parkinson’s disease for 5 or more years.

“Less than 1% of U.S. patients participate in clinical trials,” reports Dr. Kathleen Clarence-Smith, as president of ASENT, (American Society for Experimental Neurotherapeutics), a professional organization devoted to clinical research for new therapies. Recruiting for human trials participants is a major hurdle and the most frequent cause of delay in seeking FDA approval for new treatments and drugs.

Linda Herman took time off from her full time job as a librarian at Medaille College in Buffalo, New York to attend this collaborative meeting. As co-author of an invaluable resource book, When Parkinson’s Strikes Early, compiled from 70 contributors to the PARKINSN Discussion list from the University of Toronto, she is a highly regarded spokesperson for the grassroots PD community. She explains her job as a volunteer PPP Tracker explaining how she develops a relationship with the sponsor company and coordinates information input to a database designed to compile a comprehensive array of information important to the ultimate marketing of a new therapy. Her assigned scientific treatment and the 32 other PD treatments in the pipeline are each updated as they go through the required FDA phases toward approval.

Seated beside Herman is a former school principal and national advocate from Tennessee, Peggy Willocks, recipient of the Parkinson’s Action Network Grassroots Advocate of the Year. Scientists and industry produce the knowledge and develop the products. “You won’t find the cure in Washington,” she tells the illustrious group. “It all germinates at the grassroots level,” she says referring to the efforts of PPP to work with the pharmaceutical industry. She was one of six participants in a clinical trial involving experimental brain surgery for retinal cell implantation.

Bernard Brannigan from Staten Island, NY, continues, “What makes the Pipeline Project approach to tracking clinical trials unique, is that we not only track the progress of the potential treatment through the study, but we do a business assessment of the company as well. We believe a well-rounded approach is important to the Parkinson's community. This is especially important today when many of the potential treatments are in the hands of small biotech companies with limited cash. If there's a possibility that a company may not have financial resources to complete a trial, we want potential trial participants to know." Brannigan is the Tracker Coordinator for the Pipeline Project and also for Guilford Pharmaceuticals in Baltimore, who have a promising treatment in Phase II trials now.

The PPP provides the training and infrastructure needed to inform the patient advisors and help fledgling companies survive the stringent regulatory process. Sponsors of new drug trials are offered to be networked with patient advisors in “community advisory panels” to incorporate this essential perspective. Dr. Cohen, who received his Ph.D. in organizational Behavior from MIT Sloan School of Management, leads such a council for Guilford. The FDA is considering greater patient participation in its regulation of industry for neurology, much like already is done for cancer. The use of Research Partners in clinical research centers to counsel patients is an important component of the program for recruitment, protection, and retention of research settings. The Pipeliners came to Washington in search of funding and support to establish such a national network.

The concept of the Parkinson Pipeline Project is simple, but the hard work is evident in the online collection of current trials, valuable information, and links of promising drugs and treatments. The website now under development will draw on the database to provide public information and training for staff and support the other functions of the project. “There’s nothing else like it out there,” commented Jason Sullivan of the Kinetics Foundation. “It has enormous potential to become the amazon.com of clinical trials.” The highly praised web site and database was designed and is maintained by Parkinson's advocate Carolyn Stephenson, a former human resources manager, of New York State

As a patient representative for Parkinson’s at the FDA, Dr. Cohen, who was a health care consultant prior to his PD diagnosis, has been working with the Parkinson’s Disease Foundation on the concept of the PPP for several years, trying to muster up needed personnel and funding to get the project in full swing. “The most unique part of this effort is the patient component. They can obtain information and provide insight that no one else can.”

By the close of the meeting, the project received high accolades from every organization represented. The next move will be to take the information presented back to their respective groups to see what support and/or funding they can contribute. As for the five presenting their project today, they will go on coping with the degenerative disease each day feeling a little bit closer and a lot more involved in finding the cure.

 
Robin Elliott, Executive Director of PDF reported on an article this year in the Journal of the American Medical Association declaring U.S. clinical trials enterprise to be in a state of "crisis." He said, "My office has estimated that current trials . . . have more than 16,000 "slots" for study subjects . . most of which remain unfilled." He continues, "Out of this has developed a serious interest in exploring a potential role for PDF in this area . . . as a first initiative in this area, PDF is considering the development and creation of a comprehensive, interactive, user-friendly website designed to bring information on all available clinical trials to the communities of patients and families; doctors and other professionals; and the interested public." The Parkinson Pipeline Project might just be the answer.