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Connecticut Parkinson's Working Group Newsletter

July 2000

Clinical Trials                    Jackie Dorwin

"Clinical research trials." This phrase sounds so imposing. What is an ordinary fifty year old mother of three doing in one? It all started when my PD had progressed to a point where my neurologist suggested a consultation at the Yale Movement Disorders Center. The usual motor and neurological tests were done, some questions and then an evaluation. I thought that that was it, when the director of the Center joined the examining doctor and me and brought up current research, potential for a cure, and how I might become involved. Was this something I wanted to do? After we discussed the commitment, risks, and clinical trials in general I agreed. That was all there was to my getting involved in the fight to find a cure for PD.

The decision has been one of the most satisfying I have ever made. However, participating in a clinical trial is not for everyone. In a clinical trial a new drug is taken to see how your body reacts. Of course no one knows exactly what will happen (that is the point of the trial) and there might be risks. I think of the "guinea pigs" in the past who took levodopa, thank them for SinemetR, which has so improved my life, and applaud their courage. Then I think of my children: If one of them got PD would they benefit from my research participation? Since I believe that I should do what I can for as long as I can, I will do clinical trials even though my situation is, as is true for everyone with PD, deteriorating.

The FDA prescribes the steps to bring a new drug to market. This process can go on for 15 years and cost as much as $500 million. A typical case goes something like this: A research team develops a drug which they believe will benefit PWP; Animal tests are done to determine safety and efficacy; tests on humans are done. It may end at any stage if the drug proves dangerous or is not effective. The tests on humans are called clinical trials. Phase I involves fewer than 100 normal, healthy people to test the drug's safety. Phase II utilizes several hundred volunteers who have the disease to test the drug's effectiveness. Phase III can involve several thousand people with the disease and evaluates, among other things, side effects. Frequently this phase is a double-blind, where the patient is given either a placebo or the real thing, no one knows which

Even if these steps are successful, the process is not over . The pharmaceutical company proceeds with a New Drug Application and the FDA scrutinizes the drug and the research process in its entirety. Approval of the new drug means that it can be prescribed by physicians, but the studies don't end there. The company continues to submit reports to the FDA, and occasionally a Phase IV study is required

Each clinical trial has its own participation criteria . Some want newly-diagnosed and as yet unmedicated PWP; Some want those who are only on SinemetR. There may be restrictions on the length of time you have had your PD; There may be a certain symptom they are trying to target; so you must have this symptom. You must be able to follow directions and be available for appointments at specific times. An Informed Consent Form must be signed by you and witnessed; you are free to withdraw from the study at any time.

And then there are the tests. All trials start with a basic neurological exam. For the fingertapping, toe-tapping, walk-and-turn wizards that we are, this establishes a baseline from which progress can be evaluated. Blood work, EKG, urinalysis, and sometimes a cognitive function test are done. (I've pretty much memorized counting backwards from 100 by 7, and I can easily list five items in a store that begin with the letter "b").