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Risks vs. Benefits: Why I Participate in Clinical Trials

Oct 01 2007

Jolee Mohr’s family wants to know if her death was related to an experimental therapy for arthritis that she was receiving. So does the FDA, and the stakeholders of Targeted Genetics, the trial sponsor, and the participants in the same trial, as well as thousands of patients considering participation in any clinical trial. And although the latest reports indicate that the gene therapy used is not likely the blame, the results may never be 100 percent conclusive.

Mohr, 36, died at University of Chicago Medical Center on July 24, just weeks after receiving her second experimental injection of genetically altered viruses via a vector, or carrier. Traces of the gene therapy vector were found in her liver, but in predictable small amounts. Preliminary results indicate she died of massive Histoplasma capsulatum infection, a fungus infection, and possibly a lowered immune system due to an arthritis drug she was already taking. But that probably won’t ease the minds of other patients considering entering a clinical trial – any clinical trial.

For safety reasons the FDA has put the trial in which Mohr was participating on hold while the results are investigated. All other trials involving gene therapy delivered via a viral vector are on alert and have been reviewed. This has created lots of questions for those who are participating in a Parkinson’s trial using a similar virus implanted in the brains of patients. I personally know two of these participants, and they are doing well and were informed of the risks.

Seven years ago it did not take my family and me long to decide whether or not to join a trial requiring brain surgery. In August of 2000 I had experimental brain surgery for Parkinson’s disease, the second person in the world to undergo this therapy. Retinal cells, which produce dopamine, the chemical in which my brain is deficient, were implanted in an attempt to better control my Parkinson’s symptoms. Knowing I would be examined by some of the nation’s best doctors at some of the best facilities was an incentive. And I knew that Parkinson’s was progressive and had no known cure. I went into the trial thinking if the surgery did nothing but slow the progression of the disease, then it was a benefit that far outweighed the risks.

Why do people enter a clinical trial? An obvious reason is to better control a medical problem they have or find a cure. A 2005 Harris Interactive Survey of people with Parkinson’s said 95 percent agree that clinical trials are necessary in finding better treatments and 80 percent would likely participate if a trial was being conducted in their area. But more than 80 percent of clinical trials are delayed because of lack of research volunteers, with the majority of physicians who treat people with Parkinson’s referring ten percent or fewer of their patients for clinical trials. Surprisingly, it is estimated that only one percent of people with Parkinson’s living in the U.S. participate in clinical research.

Volunteers for clinical trials are led through a process known as informed consent to ensure participants understand the trial protocol, financial obligations, the time commitment, and the risks. I knew that I was risking a lot being in the first group of humans to undergo my experimental treatment. But to do nothing to try to stop this devastating disease or to provide hope for others was just as risky.

There are no failed trials – just adverse effects or a financial decision to halt a study. Just as it is with life, we learn from our mistakes. If I had learned in my trial that the patient before me had died, regardless of the reason reported, would I have gone through with the procedure? That’s a hard call and a decision each individual must make. But to let this deter others from volunteering for clinical trials is to let Jolee Mohr or others have died in vain. We don’t know what will happen when a new treatment or drug is tried on humans; but we won’t know if we don’t try.

Peggy Willocks
PAN Board member & Tennessee State Coordinator
Parkinson Pipeline Project

Source Publication: Johnson City Press