Perry D. Cohen, Ph.D., Parkinson Pipeline Project
GDNF and the Patient Perspective on New Therapy Development
A law suit has been filed in Federal court this week against Amgen by patients in the GDNF clinical trials to obtain the medication as was promised when they took the risks including brain surgery to test this experimental treatment. See the description of the "FDA patient consultant program" for PD.)
The voice of the patient is a key missing ingredient in the development of new therapies. Most researchers and sponsors of clinical trials still relate to human research subjects much the same as they relate to laboratory animals. We suggest that collaboration with patients is a more productive route to find better treatments. We have developed the “research participant’s bill of rights” to specify how to build trust in a new patient centered approach to therapy development.
This partnership and patient centered approach is especially relevant for PD. We suffer from a brain disease which affects movement and also to varying degrees affects more problematic aspects of functioning in the autonomic nervous system and the cognitive-emotional areas of the brain. Research participant have insights into effects of therapies than researchers rarely capture. Many PD patients are well educated in a variety of professions and many have reached the highest levels in their professions including leaders like the late Pope John Paul and former Attorney General, Janet Reno.
The public is beginning to lose faith in the medical miracles of the past as more and more information comes out about the unintended consequences of ingesting powerful chemicals into your body. The unraveling of the mighty pharmaceutical industry is not out of the realm of possibility. The assertion of the voice of the patient as a counter balancing force to this extreme is an important story which has not been told. Amgen provides many lessons on how to relate to patients, unfortunately most of the lessons are negative (what not to do, including their "successful" so far PR campaign to paint GDNF as unsafe and not effective).
As for all companies in the pharmaceutical industry, Amgen has benefited greatly from massive public funding of biomedical research, and in this case government grants supported successful phase I studies. The company must be publicly accountable for its actions.