pharmaceuticals

First Drug Approved for Dyskinesia in Parkinson’s Disease

Adamas Pharmaceuticals recently announced U.S. Food and Drug Administration (FDA) approval of an extended-release formulation of amantadine (GOCOVRI) to treat dyskinesia in Parkinson’s disease. This is the first drug indicated specifically for dyskinesia — uncontrolled, involuntary movements that can develop with long-term levodopa use.

Extended-release amantadine is intended to be taken once daily at bedtime. In this way it can control dyskinesia during the day, when it typically is most prevalent. The new therapy’s approval is based on data from three placebo-controlled trials that demonstrated safety and efficacy. In addition to easing dyskinesia, the drug also may lessen total daily “off” time, when Parkinson’s symptoms return because medication is not working optimally.

The Michael J. Fox Foundation (MJFF) helped move this drug to market by supporting the creation and authentication of the Unified Dyskinesia Rating Scale, a tool that was used to measure the drug’s impact in trials.

“Dyskinesia can significantly compromise quality of life for people with Parkinson’s,” says Todd Sherer, MJFF CEO. “We are pleased that patients have another option to manage this aspect of the disease and glad the Unified Dyskinesia Rating Scale — a tool our support helped develop and validate — could show clinical efficacy of GOCOVRI for the treatment of dyskinesia.”

Extended-release amantadine is a reformulation of a currently available generic immediate-release version, which is approved to treat Parkinson’s symptoms.

 

Article from Michael J. Fox Foundation for Parkinson’s Research.

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Free Water Levels Provide a New Biomarker for PD Progression

According to a recent study, a newly discovered biomarker, free water, can track changes in the brain that are associated with Parkinson’s disease, which ultimately may aid in developing new drugs that could slow disease progression.

“This finding is a potential game changer as it could shift the way Parkinson’s disease clinical trials are designed and conducted,” said Michael S. Okun, MD, a professor and chair of neurology at the University of Florida and medical director for the Parkinson’s Foundation. “Free-water is a validated measurement that will likely decrease the number of patients required to demonstrate the slowing of clinical progression.”

The study titled, “Progression marker of Parkinson’s disease: a 4-year multi-site imaging study,” was published in the journal Brain.

One of the issues in developing disease-modifying therapies for Parkinson’s disease has been a lack of an accurate biomarker that can detect changes in the brain as the disease progresses. Recently, a new imaging technique was developed that can accurately detect the volume of water in brain tissue and separate that measurement from the water outside the brain tissue. The latter type of water is known as free water and has been known to increase in neurodegenerative disorders.

In 2015, researchers demonstrated that free water levels were increased in the posterior substantia nigra (PSN) of patients with Parkinson’s disease. The motor symptoms that accompany a diagnosis of Parkinson’s disease tend to emanate from the area of the brain that includes the nigrostriatal pathway, which is part of PSN.

In another study, researchers discovered that the free water levels in the PSN increased over one year in newly diagnosed Parkinson’s disease patients, but not in control groups. However, no studies have investigated how free water in the PSN changes over an extended period of time.

Therefore, researchers at the University of Florida conducted a multicenter international longitudinal study to determine the pattern of change in free water in patients with Parkinson’s disease over four years.

Results from this study showed that free water levels in PSN increased over one year in newly diagnosed Parkinson’s disease patients. Furthermore, free water levels continuously increased over four years. The research team also showed that sex and baseline free water predicted four-year changes in free water levels. Additionally, researchers showed that free water increasing over one or two years leads to worsening stages on the Hoehn and Yahr scale over a four-year period.

One of the most important things to result from this study has been the discovery of a biomarker that determines the progression of Parkinson’s disease and one that can potentially be used in future clinical trials as an endpoint.

“This means if you want to start designing studies to slow the progression of Parkinson’s disease, testing a drug on that measurement in the substantia nigra might be a good way to go,” said David Vaillancourt, PhD, professor of applied physiology and kinesiology at the University of Florida in a press release. “If the measurement in the substantia nigra is increasing year after year after year, and if you can stop that from occurring, you’re likely to slow or possibly stop the progression of the disease. This could change the way studies are conducted for disease-modifying trials in Parkinson’s disease.”

 

Article from Parkinson’s News Today.

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31st Annual Parkinson Disease Symposium | June 23, 2017

WPA will host its 31st Annual Parkinson Disease Symposium on Friday, June 23, 2017 at Country Springs Hotel & Conference Center in Pewaukee, Wisconsin.

Beginning at 8:00am, attendees can check in and visit with vendors at the Resource Fair where health and community agencies will display valuable information throughout the day. Beginning at 9:00am, the first main session will be “Understanding Parkinson Disease from a Scientific Perspective”, presented by Giuseppe P. Cortese, PhD, Postdoctoral Research Associate, Department of Neurology, University of Wisconsin-Madison. The morning breakout sessions will follow Dr. Cortese’s interactive presentation, and participants will choose from three options: “Caregivers: Being prepared for an emergency”, “Grieving ‘life as we have known it’”, and a Panel on PD exercise programs.

During lunch, the resource fair will again be open for participants. After lunch, the afternoon breakout sessions will include “Are you caring too much and laughing too little?”, “Causes and prevention of falls” and “Exercise: A targeted attack on Parkinson’s.” The closing session for all attendees will be “Nutrition for Parkinson Disease” presented by Michelle McDonagh, RD, CD, Froedtert & The Medical College of Wisconsin. The Symposium will conclude by 3:30pm.

The registration fee is $30 per person and includes educational materials, continental breakfast, and lunch. To register, CLICK HERE or call our office at 414-312-6990. Registration is required and must be received by Wednesday, June 14.

The event is sponsored by Abbvie, Medtronic and US WorldMeds.

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FDA approves drug to treat Parkinson’s disease

The U.S. Food and Drug Administration recently approved Xadago (safinamide) tablets as an add-on treatment for patients with Parkinson’s disease who are currently taking levodopa/carbidopa and experiencing “off” episodes. An “off” episode is a time when a patient’s medications are not working well, causing an increase in Parkinson’s symptoms, such as tremor and difficulty walking.

“Parkinson’s is a relentless disease without a cure,” said Eric Bastings, M.D., deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “We are committed to helping make additional treatments for Parkinson’s disease available to patients.”

The efficacy of Xadago in treating Parkinson’s disease was shown in a clinical trial of 645 participants who were also taking levodopa and were experiencing “off” time. Those receiving Xadago experienced more beneficial “on” time, a time when Parkinson’s symptoms are reduced, without troublesome uncontrolled involuntary movement (dyskinesia), compared to those receiving a placebo. The increase in “on” time was accompanied by a reduction in “off” time and better scores on a measure of motor function assessed during “on” time than before treatment.

In another clinical trial of 549 participants, the participants adding Xadago to their levodopa treatment had more “on” time without troublesome uncontrolled involuntary movement compared to those taking a placebo, and also had better scores on a measure of motor function assessed during “on” time than before treatment.

The FDA granted approval of Xadago to Newron Pharmaceuticals.

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm547852.htm

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