Episode 108: The Golden Year for Testing Disease Modifying Drugs

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.

Released: July 13, 2021