Why should you participate in Clinical Trials for Parkinson’s?

It’s so crucial that patients with Parkinson’s be part of the solution, that they participate in trials and help move the ball forward so that people like you …. can benefit from the knowledge that we get from these trials.”

Andrew Siderowf, MD

You do trials for yourself because you hope it works, and then two to three years down the line it may work and it is for you.

Susan Lehman

Did you know that most clinical trials cannot be completed because researchers cannot recruit enough people to participate?

If you are like most people, you are wary about participating in Clinical Trials, especially if they involve medication.  I have shied away from drug trials.  My excuses range from “I don’t want to take the risk with an unknown drug” to “I don’t want to go off of the medications that are working for me”.  Or “I need to check with my doctor” which, of course, I never do.  However, I have taken numerous surveys and participated in several cognition and exercise trials, which are also important.

The latest Sunday Mornings with Twitchy Woman program featured two of our women with Parkinson’s, Lauren Simmons and Susan Lehman, who spoke about their recent experiences with drug trials.  Andrew Siderowf, MD, the Hurtig-Stern Professor of Neurology and the Chief of the Movement Disorders Division in the Penn Perelman School of Medicine Department of Neurology, and Ellen Indik, Clinical Research Coordinator at Penn, spoke from the researcher’s point of view.

Different types of Clinical Trials

We learned that there are 3 different types of Clinical Trials

  1. Observational trials – filling out surveys, not invasive at all
  2. Interventional studies – give a blood sample or spinal fluid sample in addition to surveys. The Fox Foundation PPMI study is a good example. 
  3. Interventional drug treatment studies.  
    • Discovery Phase (Phase 1 or 2).  The goal is to see if it is safe, what the right dose is, and what the spectrum of possible side effects are.  These are often very intensive trials, especially for the patients.  In addition, researchers learn how to use the drug before going on to bigger trials. These trials are often very short, maybe 2 months long.  They often have limited patients on placebos so that they can understand more about the drug.  Usually there is a 6:1 or 4:1 ratio of drug to placebo.
    • Confirmatory phase.  These are the big studies that show the FDA that the drug is safe and effective and can be approved to take to market.  They often involved 100s of people in multiple places in the country.  2:1 or 1:1 placebo rates are used to help confirm the earlier findings.  These studies are often longer, a year or more, and can be extended so that people who were on the placebo can be treated with the drug in the second half of the trial.  This is a lower risk activity for the patients.

Dr. Siderowf recommends that you talk to your family and doctor before agreeing to a drug trial. If you can, say yes, because this is the way we are going to beat Parkinson’s.

The Patient’s Experience

Susan and Lauren had very different experiences with drug trials.  Susan’s was positive.  She says that the good news is that she thinks the drug worked.  The bad news is that it is no longer available and she hopes it will become available to her soon.  She wants to be a part of the solution, not the problem.   

Lauren’s experience with a pump delivery system for Carbidopa/Levadopa was not so positive.  She had problems with the pump from day one, with bruising and swelling.  She is very active and had issues with placement during exercise.  When it worked, she felt great, with no off times.  Unfortunately the physical issues with the pump forced her to withdraw early from the study.  However, she says, she thinks that they learned a lot from the problems that she was having.

If you are interested in learning more, watch the video from the meeting.

Hopefully you will find that you also want to be part of the solution to Parkinson’s and sign up for some future trials. Check with your doctor to see what is available in your area or sign up on the Fox Trial Finder site to be matched with appropriate studies. You can also go the the NIH website to learn about other Parkinson’s trials.


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A Note To My Readers

I love to see your comments and get your emails as we share our collective experiences. But based on a couple of private questions from some of you, remember, I am just a lay person and a patient like the rest of you. For medical and similar advice, you need to talk to your own doctor

Twitchy Woman

Twitchy Women partners with the Parkinson’s Wellness Fund to ensure we have the resources to offer peer support for women with Parkinson’s.