In this issue of The Doctor Is In, we are focusing on Clinical Trials at The Parkinson’s Institute. We are a world leader in evaluating new potential therapies and diagnostic tools, and we know that with the help of clinical trials, we can get answers to questions that bring us closer to better treatments and a cure.
Often our clinicians are asked why we conduct Clinical Trials. At The Parkinson’s Institute, we target many realms of Parkinson’s disease, from understanding it biologically to treating PD patients on a daily basis. As experts on Parkinson’s disease, we strive to be at the forefront of developing and evaluating new medications, new treatments and new efforts to manage this disease. Conducting clinical trials is one of the critical ways we achieve this goal, and is how breakthroughs in science are brought to the patient. Through clinical trials, we gain much crucial understanding about the disease that cannot be gained any other way.
What goes on during a clinical trial? Some clinical trials are observational in nature, for example, evaluating changes that occur in PD as it progresses, or looking for new markers or tests that can inform us about how to better diagnose the disease or follow progression. Many clinical trials are done to test the safety and efficacy of new or existing drugs. Other trials involve testing non-drug therapies and innovative medical devices. On occasion, we also have studies that need participation from people without PD.
We have a resident expert, Dr. Grace Liang, answering questions about clinical trials sent in by people like you. Dr. Liang is a movement disorder specialist and has her own clinical practice. Liang is the Director of Clinical Trials, and has been with The Parkinson’s Institute since 2005.
Q. Can you please explain what the clinical trial process is?
When new drugs or devices are being evaluated as potential treatments, clinical trials must be performed to answer two critical questions: Is the treatment safe and tolerable to use in the people it is intended for? Is it clearly effective in providing the benefit or improvement it is supposed to for the condition?
Clinical trials are performed in various phases for approval of a treatment in the US by the Food and Drug Administration (FDA).
Phase I are the first studies done in healthy human subjects after careful laboratory and animal tests have been completed, and involve small numbers of individuals to be able to get initial information on dose ranges that are safe to use and how the drug is metabolized or processed by the body.
Phase II studies involve several dozen subjects, and are tests in individuals with the disease or condition targeted, to gather more data on safety and tolerability of the treatment, dosing regimens that will be used, and initial tests of if the treatment is effective.
Phase III studies are large-scale, usually multi-center studies involving hundreds to thousands of patients, and are the "pivotal" studies that must be positive to prove safety and efficacy of the treatment for FDA approval.
After studies are approved, sometimes Phase IV studies are conducted to further evaluate the treatment for new uses or to study long-term effects. This process generally takes many years to complete.
There are many levels of monitoring and protection to ensure as best as possible that the studies are performed with the highest quality of scientific standards, ethically, and safely for all the volunteer subjects involved. The doctors and staff involved in conducting the study examine and monitor each individual with many tests as the study is being performed, and there are external auditors, expert panels and reviewers overseeing the trial at every step. Sometimes, if it is shown that there are side effects or risks of a treatment that outweigh the benefits, or if a treatment is shown to be clearly ineffective, the trial may be stopped early. If the study goes as planned, the FDA reviews all of the data gathered from all of the studies done to make the final determination of whether it is a worthwhile treatment to approve for release.
Q. If I’m in a clinical trial, how can I be sure that I’m getting medication that will help me and not hurt me?
Many clinical trials are designed to answer the question: Is using this new treatment as good as or better than what is currently available, or better than doing nothing? While that seems like a simple question, it is often not that easy to answer. For this reason, many studies are “randomized controlled trials,” where the new experimental treatment is compared to either a known treatment or to a “placebo” (a pill that looks the same but has no active drug in it). Often, the individual and the staff at the sites conducting the study are “blinded,” meaning that they are not informed of whether they are getting the active treatment or not. This is necessary to reduce any bias or prejudice in doing the evaluations, because many times just thinking that you are being treated with something can cause a change in symptoms or appear to cause improvement. Sometimes, trials are “open-label,” where you know for sure you are getting the actual drug.
Because participating in a clinical trial is completely voluntary, it is our job to inform volunteer subjects prior to participation, of any potential risks, discomforts, or benefits of participating in the trial, so that they can make an informed decision. A person can decide to stop participating in the trial at any time for any reason as well. Every individual’s safety and health is our number one priority, and so we take extremely careful measures to monitor an individual throughout the trial through frequent exams, laboratory and other tests. Many people find that in participating in a clinical trial, they are able to interact much more with the doctors and staff, and learn a great deal about Parkinson’s disease and their own health.
Q. Is there anything that can be done to expedite bringing new PD drugs into the market?
It is essential that people with Parkinson’s disease stay informed and educated about new developments in the field, and to continue supporting research both at a local community level, as well as on a broader scale, making people aware of the necessity of research for Parkinson’s disease. The process to bring a new potential treatment to market takes many years, from start to final approval. One of the major hurdles in this process is that it is often slowed down by difficulty recruiting enough patients into the trials. If possible, consider volunteering as a participant in a clinical trial. Without the willingness and commitment of thousands and thousands of individuals to help in this effort, no new treatments or drugs would EVER be available for the benefit of all. As one volunteer said, “We are not guinea pigs—we’re heroes!”
Most Clinical Trials cover the cost of your visits, exams and lab tests performed related to the study, as well as the study drug if there is one. Some trials will also help cover the cost of transportation and/or accommodations. If you would like to be involved in one of our Clinical Trials, visit our Clinical Trial Volunteer form or call the Clinical Trials number at (408) 542-5608, and a Clinical Trials Coordinator will contact you.