Podcast Episode 108: The Golden Year for Testing Disease Modifying Drugs

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Most people with Parkinson’s disease (PD) first seek medical care when they recognize or are troubled by symptoms – often stiffness, slowness of movement, or tremor. They may go on drug therapy at the time of diagnosis or, typically, within six to twelve months to relieve those symptoms.

In order for researchers and drug developers to test and ultimately find drugs that can slow the progression of the disease, they need to test those drugs in people who are not already on medications to alleviate symptoms and compare them to similar people taking a placebo. Currently there is no blood test or other biomarker to measure progression, so the most common and straightforward way for drug trials to judge progression is to observe signs and symptoms in people not receiving symptomatic medications such as levodopa, dopamine agonists, or other drugs that make symptoms less apparent.

Dr. Robert Hauser, Director of the Parkinson’s Disease and Movement Disorders Center at the University of South Florida in Tampa, urges people early in the course of their disease to enter a clinical trial as soon as they receive a PD diagnosis and before they go on medication. The longer that they can be observed before taking a potential disease-modifying medication, the greater the ability of researchers to detect changes, or in the case of a drug in development, if successful, not to see changes. However, patients who can go up to a year without medication are in short supply, in part because patients often wait to seek medical help until they want medication. As Dr. Hauser wrote in a medical publication a few years ago, “… why see a doctor when you don’t need treatment?”

But the answer is because people very early in the course of their disease are vital to finding drugs that can slow progression. Dr. Hauser terms the critical period of about one year between the time of first motor symptoms appearing to initiating symptomatic therapy the “Golden Year” for participation in disease modifying clinical trials. So, he wrote, “It is critical that care providers and patients don’t unknowingly waste this golden year.” In this episode he lays out the problem, has messages for patients and providers, and tells how such a system of referral to entry into clinical trials can work.

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About This Episode

Released: July 13, 2021

Robert A. Hauser, MD, MBA, FAANHeadshot of Dr. Robert Hauser

Dr. Robert Hauser is a Professor of Neurology at the University of South Florida College of Medicine, in Tampa, Florida. He serves as Director of the USF Parkinson’s Disease and Movement Disorders Center, a Parkinson Foundation Center of Excellence. Dr. Hauser earned his medical degree from Temple University School of Medicine in Philadelphia, Pennsylvania, and completed neurology training at the Eastern Virginia Graduate School of Medicine, in Norfolk, Virginia.

Dr. Hauser completed a fellowship in Movement Disorders at the University of South Florida and became Center Director in 1994. He has authored or co-authored more than 300 peer-reviewed publications and is one of the world’s most cited Parkinson’s Disease investigators. He is Past Chairman of the Interventional Neurology Section of the American Academy of Neurology, has served on the executive committee of the Parkinson Study Group, and was a member of the steering committee for the NIH-sponsored Neuroprotective Exploratory Trials in Parkinson’s Disease program (NET-PD). Dr. Hauser lectures frequently at scientific meetings and served as Chairman of the 2009 World Federation of Neurology International Congress on Parkinson’s Disease and Related Disorders. 

Dr. Hauser’s primary research interest is the development of new medical and surgical treatments for Parkinson’s disease and other movement disorders. He has extensive expertise in clinical trial design and execution. The outcome measures he developed have become the gold standard for use in clinical trials. He maintains an active patient practice and has been voted a Top Doctor by his peers every year since 1993.

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For all of our Substantial Matters podcast episodes, visit parkinson.org/podcast.

For more insights on this topic, listen to our podcast episode “Repurposing Medications to Slow Progression of PD.”


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