Michael J. Fox Foundation-funded Study Identifies Best Clinical Rating Scale
Researchers funded by The Michael J. Fox Foundation (MJFF) have identified the best measure for testing patient response to therapies to treat dyskinesia, the debilitating, uncontrollable movements that are often a side effect of Parkinson’s disease (PD) drugs. MJFF has made dyskinesia a top research priority due to its significant negative impact on patients’ quality of life.
The findings, from a team of researchers at eight sites across the United States, Canada and Europe, show that the Unified Dyskinesia Rating Scale (UDysRS) is superior to other tested scales for most sensitively tracking treatment effect. The study also quantified the therapeutic effect of amantadine, a drug commonly used off-label to alleviate dyskinesia, as a baseline by which to measure future potential treatments. Christopher Goetz, MD, director of the Parkinson’s and Movement Disorders Center and Glenn Stebbins, PhD, professor of neurological sciences at Rush University Medical Center, were co- principal investigators on the study.
“Historically, significant problems in measuring dyskinesia and assessing the efficacy of potential new therapies have created roadblocks to clear-cut trial results and, therefore, industry investment leading to new and better treatments,” said Alison Urkowitz, vice president of research operations at MJFF, a co-author on the study. “The establishment of the Unified Dyskinesia Rating Scale as a validated tool for use in dyskinesia clinical trials could have important implications for such investment moving forward. Now, researchers can better design accurate clinical trials to verify that a dyskinesia drug is or isn’t working.”
“Dyskinesia can be a significant problem for people with Parkinson’s disease and there is currently no therapy approved by the U.S. Food and Drug Administration (FDA) to treat it,” said Stebbins. “But there has been a big push over the past few years in both academia and the pharmacological industry to develop drugs to treat this side effect. We’re hopeful that the UDysRS will be a useful tool to help translate research into treatments.”
“We feel that this study will establish one standard scale for the assessment of dyskinesia in Parkinson’s disease, allowing one primary measurement tool to be used internationally,” added Goetz.
To date, researchers have used multiple scales to track treatment effect in dyskinesia, employing various methods: Some are based on physician-driven patient interviews, others on patient self-reporting, while others require in-the-clinic observations (UDysRS incorporates all three of these techniques). Prior to this study, there was no real evidence as to which scale provided the most sensitive measure. It was also unclear if any could provide a useful measurement on the effect of a potential drug.
In addition to showing UDysRS’ superiority, the double-blind, placebo-controlled study established effect size relative to use of amantadine, a drug that was originally approved to treat a certain type of influenza and that has been deemed efficacious for dyskinesia by the Movement Disorder Society. But this conclusion was based on small-scale studies; more research is needed to better understand its therapeutic benefit on dyskinesia. The reality is, amantadine works only for some patients and for a limited time. This study establishes a minimum benchmark by which to measure potential therapies moving forward – new drugs would hope to show better efficacy than amantadine.
Study results are being presented at this June’s International Congress of Parkinson's Disease and Movement Disorders in Dublin, Ireland. To learn more about the study’s findings, read the Foundation’s News in Context interview with Dr. Stebbins and Scientific Advisory Board member David Weiner, MD at http://www.michaeljfox.org/living_viewpoints_newsInContext_article.cfm?ID=40.
Dyskinesia is a distressing problem for many people with PD, who report that it is one of the most difficult aspects of the disease to manage. Because they fear developing dyskinesia, many patients wait as long as possible to begin using the drug levodopa, the gold-standard treatment for relieving the stiffness, tremors, and rigidity that are the cardinal features of PD. Even after starting levodopa, many limit the dosage to reduce the dyskinesia, and therefore settle for a sub-optimal benefit from the best medical therapy available for their disease.
About The Michael J. Fox Foundation
As the world’s largest private funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $289 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. Now through December 31, 2012, all new and increased giving to The Michael J. Fox Foundation, as well as gifts from donors who have not given since 2010 or earlier, will be matched on a dollar-for-dollar basis with the $50-million Brin Wojcicki Challenge, launched by Sergey Brin and Anne Wojcicki.
About Rush University Medical Center
Rush is a not-for-profit academic medical center comprising Rush University Medical Center, Rush University, Rush Oak Park Hospital and Rush Health.
In January 2012, Rush opened a new 14-floor, 806,000-square-foot hospital building near the corner of Ashland Avenue and the Eisenhower Expressway. The new hospital, is the centerpiece of a ten-year, $1 billion campus redevelopment plan called the Rush Transformation, which also includes completion of a new orthopedics building, a new parking garage and central power plant, renovations of selected existing buildings and demolition of obsolete buildings. The new hospital is designed and built to conserve energy and water, reduce waste and use sustainable building materials. The new hospital has earned LEED Gold certification from the U.S. Green Building Council and verified by the Green Building Certification Institute. It is the largest new construction healthcare project in the world to be LEED Gold certified. It is the only full-service green hospital in Chicago.
Rush’s mission is to provide the best possible care for its patients. Educating tomorrow’s health care professional, researching new and more advanced treatment options, transforming its facilities and investing in new technologies—all are undertaken with the drive to improve patient care now, and for the future.