Post Written by Serena Hung, MD,
Before I launch into my post about research, I wanted to acknowledge that April is national Parkinson's Awareness Month. I think a good summation of all of our efforts was put forth by Nevada Sen. Harry Reid in a statement: "We must build on our commitment to accelerate and increase federal support for Parkinson's research so that we can find a cure for this disease and end the hardships for the more than one million Americans who suffer from this disease and for their families." To read the full statement and for a great resource of information, visit www.parkinson.org
Now, about research ...
Mr. B is a patient who I recently saw for the first time for Parkinson’s disease (PD). After confirming the diagnosis and discussing various aspects of the disease including treatment options, he said, “One of the reasons I decided to come here is due to my brother’s urging. He is a doctor out west and he recommends seeing someone at an academic medical center because doctors at academic medical centers tend to keep up with research. What can you tell me about research in PD and am I a candidate to participate?” I haven’t been asked this question in a while and I was pleased to answer. I’ll share my answer in two parts. I’ll speak about the various research projects we have here at the Parkinson’s and Movement Disorders Program
in a later blog. Today I thought I’d talk about what it means to be in a research study.
Let me back up a little bit. Why is research needed? It may seem apparent to a lot of people. We are a lot farther in understanding a lot of diseases, Parkinson’s included, but we are not quite where we want to be. People are still suffering. In order to advance understanding and treatment of the disease, research is needed. Research comes in many forms. Some occurs in laboratories with cell cultures, chemical compounds, etc. Some involves animals such as flies, mice, primates. We call those pre-clinical studies. Those are done to collect as much data as researchers possibly can, especially safety data, before testing anything on humans. There are government and local institute (e.g. university research institutes, medical schools) regulations in place so that the least amount of foreseeable harm will happen to the human research volunteers.
Then the human participation starts. There are two major kinds of research involving human volunteers. One is called observational trials — volunteers are treated how they normally would be by their own doctors, but they come in periodically for testing (could be a physical examination, blood and urine tests, or imaging studies). The purpose of observational studies is to gain more knowledge about the natural course of the disease. The other kind of studies are treatment trials — volunteers are given a treatment and tests are done to evaluate how well this treatment works.
Why do people participate in research? I have heard a lot of different answers, and as a prior research volunteer myself, I can think of a few as well. The reasons for my participation was part financial (think of the options available for a poor cash-starved medical student; the studies I participated in reimbursed me for the time I spent in the laboratory and travel expenses). More important, I wanted to help. Being a medical student at that time, I was starting to understand what it took for knowledge to come about. No matter how good the animal models are, they are not human and those results may be quite far from what really happens when the same treatment is given to humans. I wanted to do my part (at that time, I wasn’t yet skilled enough to conduct research myself). My patients now tell me the same — they want to help. Depending on the specific aim of the study, some patients may not derive benefit for themselves right away (some may) but advancing science and potentially helping other people who suffer the same disease is one of their goals and sometimes is the reason driving them to participate in clinical research.
When thinking about participating in clinical research, there are few factors I think patients and their families should keep in mind. One, are they comfortable with the idea of research in general? Keep in mind that research is used to gather information. In observational trials, there is no drug or treatment given, hence no “treatment benefit” is expected. In treatment trials, the treatment in question is not an established therapy. That means whether it is helpful or not remains to be seen. Also, are they comfortable with the possibility that they may be in the group called the “placebo” or “control” group? In some trials, the placebo group gets, essentially, a sugar pill and neither the patients nor the researchers would know what kind of pill the patients are getting until after the trial finishes (this is called “blinding” and is used to avoid bias). In some trials, the control group still gets the treatment, but may get it a few weeks or a few months later than the rest of the patients in the trial. Once again, the researchers do not know what the patients in the trial are receiving. Since a research study is done to find out information, sometimes there are side effects that were not known until the treatment is given to humans.
There have been studies consistently saying that patients who participate in clinical trials tend to do better and feel better overall, even if they happen to be in the placebo or control group. The explanation for this is not clear. It could be that having follow-up appointments with physicians more frequently is helping (during the research study, typically the duration between follow-up appointments is shorter). It could also be that participating in clinical trials is motivating in some ways. For me, I am just happy that some patients think about research and want to participate since new developments are always exciting, and without patient participation, we will never get the knowledge we want and need.
Back to Mr. B — we talked about all the trials that we have ongoing at Froedtert & The Medical College of Wisconsin. Since he is newly diagnosed and hasn’t been on any Parkinson medications yet, he is perfect for one of the studies about slowing down the progression of the disease. He is currently thinking about it, and will let me know soon.