The time between diagnosis and the beginning of symptomatic treatment is critical in the effort to find a cure for Parkinson’s disease (PD). A paper published in npg Parkinson’s Disease, a Nature Partner journal, notes too many early PD patients wait too long before seeking medical attention, or start taking symptomatic medications before they are required, thereby dramatically shrinking the pool of candidates for clinical trials.

Parkinson’s disease is a disorder of the central nervous system that affects movement. Symptoms include tremors, stiffness, and slow and small movements. The pace of progression varies among patients, making the months following diagnosis crucial to researchers studying the disease’s progression.

Robert Hauser, MD

“The critical time of about one year from when the patient can be diagnosed with early PD based on mild classic motor features until they truly require symptomatic therapy can be considered the Golden Year,” said lead author Robert A. Hauser, MD, director of the Parkinson’s Disease & Movement Disorder Center, based at the USF Health Neuroscience Institute. “It is during this early, untreated phase, that progression of clinical symptoms reflects the progression of the underlying disease.”

Dr. Hauser says that in order to determine whether or not a potential disease slowing therapy is actually working, clinical researchers must be able to compare the therapy to a placebo without interference from symptomatic treatment. Otherwise, they won’t know if the therapy is slowing the disease’s progression or if they are just seeing the effects of symptomatic treatment.

This requires patients to seek assessment soon after they notice the onset of tremor or slow movement. In addition, physicians should consider referring patients to clinical trials soon after diagnosis and delay prescribing symptomatic medication until it’s needed. If a patient waits until symptomatic treatment is necessary, the opportunity to participate in these crucial clinical trials is lost.

USF professor leads study on valbenazine’s effects on tardive dyskinesia. 

Antipsychotic treatment can cause involuntary movements such as lip smacking, tongue protrusions and excessive eye blinking. These movements typically  occur after more than three months of treatment and are called tardive dyskinesia.

Robert A. Hauser, MD, MBA, professor of neurology at the USF Health Morsani College of Medicine, is the lead author of a study recently published in the American Journal of Psychiatry that concludes valbenazine administered once daily can significantly reduce tardive dyskinesia in patients with schizophrenia, schizoaffective disorder and mood disorder.

Robert Hauser, MD

“One approach to managing tardive dyskinesia is to discontinue antipsychotic treatment or reduce the dosage, but these options are not always feasible, because withdrawal can exacerbate tardive dyskinesia symptoms or have a negative impact on psychiatric status. Moreover, tardive dyskinesia symptoms often persist even after discontinuation or dosage reduction,” wrote Dr. Hauser, who directs the Parkinson’s Disease and Movement Disorders Center at USF.

Valbenazine is a selective vesicular monoamine transporter 2 inhibitor. Two hundred twenty-five people with schizophrenia, schizoaffective disorder or a mood disorder participated in the phase 3 randomized double-blind, placebo-controlled trial.

Forty-percent of those who received valbenazine 80mg/day improved by at least 50 percent. That’s compared to just 8 percent in the placebo group.

Researchers also determined that valbenazine was well tolerated. Drowsiness, restlessness and dry mouth were reported as adverse effects.

Pharma, biotech leaders will discuss several new clinical studies testing drugs and gene therapy for Friedreich’s ataxia

//www.youtube.com/watch?v=EIMrtGI8Kfo

Tampa, FL (Aug. 31, 2015) — The University of South Florida (USF) will again bring together leading researchers and patients searching for a treatment for Friedreich’s ataxia and related disorders at the seventh annual scientific symposium “Understanding Energy for A Cure.”  The symposium will be held 5 to 8:30 p.m., Thursday, Sept. 17, at the USF Marshall Student Center Ballroom, USF Cedar Circle, Tampa, FL  33620.

The event, free and open to the public, is hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the USF Ataxia Research Center.

For the first time, the symposium will include a panel discussion with several biotechnology and pharmaceutical industry leaders about new clinical trials testing drugs and gene therapy for Friedreich’s ataxia. The panelists include representatives from Agilis Biotherapeutics, LLC; Horizon Pharma, plc; Reata Pharmaceuticals Inc; and Retrotope, Inc. The companies collaborate with FARA and academic institutions to focus on research that will improve the quality and length of life for those diagnosed with Friedreich’s ataxia and lead to treatments that eliminate symptoms.

Patients with Friedreich’s ataxia and their families come to USF from across the country to share their thoughts and perspectives about energizing the search for a cure.  The event will also be attended by supporters of the FARA Energy Ball gala, held on Saturday, Sept. 19.

The scientific symposium will again be broadcast through Ustream’s CureFA channel, with opportunities for visitors to join the conversation long distance. To watch the presentations in real-time, visit http://www.ustream.tv/channel/curefa on Sept. 17 at 6 p.m. EST.  A Ustream account/membership is not needed to join.

Friedreich’s ataxia is a rare, progressive neurodegenerative disease affecting children and adults for which there is currently no approved therapy. Symptoms include neurologic, cardiac, orthopedic, and endocrine dysfunction.

The symposium will be hosted by Clifton Gooch, MD, professor and chair of the Department of Neurology in the USF Health Morsani College of Medicine. Theresa Zesiewicz, MD, professor of neurology and director of the USF Ataxia Research Center, will update attendees on the Friedreich’s ataxia initiatives at USF, one of 10 sites in the international FARA Collaborative Clinical Research Network.

FARA President Ron Bartek and Jennifer Farmer, FARA Executive Director, will give an overview of progress nationwide in Friedreich’s ataxia research.

Featured speaker Sanjay Bidichandani, MBBS, PhD, chair of pediatric medical genetics and professor of pediatrics and biochemistry and molecular biology at the University of Oklahoma College of Medicine and member of the FARA Board of Directors, will talk about the pipeline of investigational treatments for Friedreich’s ataxia.  He was part of the group that discovered the gene for Friedreich’s ataxia and, over the last 15 years, his research has helped characterize the disease’s genetic and epigenetic defect.

USF Health’s Dr. Gooch will moderate a question-and-answer session on patients’ perspectives of living with Friedreich’s ataxia and clinical trial participation.

For more information, please visit http://www.curefa.org/energyball, or call (813) 974-5909.

– About USF Health –

USF Health’s mission is to envision and implement the future of health. It is the partnership of the USF Health Morsani College of Medicine, the College of Nursing, the College of Public Health, the College of Pharmacy, the School of Physical Therapy and Rehabilitation Sciences, and the USF Physicians Group. USF Health is an integral part of the University of South Florida, a high-impact, global research university dedicated to student success. For more information, visit www.health.usf.edu

– About The Friedreich’s Ataxia Research Alliance (FARA) –

FARA is a non-profit organization dedicated to curing FA through research. FARA grants and activities provide support for basic and translational FA research, pharmaceutical/biotech drug development, clinical trials, and scientific conferences. For more information, go to www.curefa.org.

Media contact:
Anne DeLotto Baier, USF Health Communications
abaier@health.usf.edu or (813) 974-3303

Video by Sandra Roa, USF Health Communications

The fundraiser teams patient with USF neurologist who studies new treatments for the neurodegenerative disease

Tampa, FL (Feb. 18, 2015) – The first time his right arm froze up, Rick Karczewski let it pass.

Then six months later on Memorial Day, driving his Harley-Davidson motorcycle up Interstate 75 to meet fellow military veterans heading to Florida National Cemetery in Bushnell, his hands began to cramp. “There’s something wrong with me,” he thought. Karczewski told his buddies he’d better head back home.

His doctor referred him to Dr. Theresa Zesiewicz, a neurologist at the University of South Florida’s Parkinson’s Disease and Ataxia Center. A battery of exams eventually confirmed his suspicions: he had Parkinson’s disease.

USF neurologist Dr. Theresa Zesiewicz (on motorcycle) will participate in the motorcyle ride benefitting the USF Parkinson’s disease center named in honor of her mother, who died from complications of the neurodegenerative disease.  One of Dr. Zesiewicz’s patients helped organize the fundraiser.

Now 18 months later, Karczewski, 49, hopes to give something back to the doctor who sat with him during those first long visits talking through all of his questions, and who has continued to treat him ever since.

On Saturday, Feb.21, the inaugural Shakes, Rattles, Rides and Rods fundraiser will be held in Riverview, aiming to raise at least $10,000 for the Frances J. Zesiewicz Center for Parkinson’s Disease at USF, as well as the Michael J. Fox Foundation.

The Frances J. Zesiewicz Center was named in honor of Dr. Zesiewicz’s mother, who died from complications from the disease.

For Karczewski, the fundraiser and ride is a way for him to combine his passion for motorcycles with his desire to contribute to the center and respond to the disease in a positive manner that can help others.

“I’d rather be proactive instead of being down on myself that I have it,” Karczewski said. He added that 100 percent of the proceeds will go to the two foundations, with most of that geared for the USF center to give back to Dr. Zesiewicz for everything she’s done for him. He recalled that for his first office visit with her, she spent close to 45 minutes talking with him.

“When she’s with a patient, that patient gets her time, no matter how long it is,” he said.

Dr. Zesiewicz empathizes with the pain and frustration of patients. She had been a USF fellow in Movement Disorders in the USF Department of Neurology for about a year in 1994 when she was out at a restaurant with her mother. As they were dining, she saw her mother’s hands shaking. “What’s that?” Dr. Zesiewicz asked her. She went on to diagnose her mother with Parkinson’s disease. Frances Zesiewicz died 16 years later in 2010, followed by her brother – Dr. Zesiewicz’s uncle – nine weeks later from the same disease.

“I have a personal stake in this disease,” Dr. Zesiewicz said.

Parkinson’s disease is a neurodegenerative brain disorder that typically progresses slowly from slight tremors to impaired motor dexterity and life-changing problems, including alteration in balance and non-motor secondary symptoms such as depression and anxiety. At least one million people in the United States live with Parkinson’s disease. There is no known cure, though treatments such as medication and surgery are used to manage symptoms.

Dr. Zesiewicz is a tenured USF professor of neurology, as well as director of the USF Ataxia Research Center, the Frances J. Zesiewicz Center and Foundation for Parkinson’s Disease at USF, and the James A. Haley Veterans’ Administration Parkinson’s Disease Clinic.

Her research team has focused on motor and non-motor symptoms of neurological diseases and the testing of new medications to treat them, including currently a national clinical trial testing a powerful antioxidant medication in patients with Friedreich’s ataxia, a rare debilitating neuromuscular disease. This medication is also being tested in Parkinson’s disease.

Dr. Zesiewicz hopes to use donations from the upcoming fundraiser for physical therapy and tai chi classes to help Parkinson’s disease patients develop core strength to counteract problems with balance that can set in as the disorder advances.

When she heard about Karczewski’s plans for the fundraiser and that he and his fellow organizers, wife and support team planned to steer donations toward the center named for her mother, she felt extremely grateful.

“They have just been fantastic in raising money and awareness for this disease,” she said. “They decided to do it and never looked back. They are giving and loving people.”

Karczewski organized the fundraising event with Mike Savidge, publisher of Go For A Ride Magazine, after discovering that Savidge’s wife, Charlette, also has the disease. The two combined forces to aid efforts to develop further treatments and a cure for the disease.

Karczewski, who started riding minibikes when he was 8 years old, said he realizes he probably only has another year left to enjoy his passion. He already has downsized from a Harley-Davidson Electra Glide to a Softail. He can ride only about 40 to 50 miles at a time before his body gets too fatigued and he has to stop.

But he always plans to play a part in the fundraisers, which he says will become annual events.

For the first one, he expects to put Dr. Zesiewicz on the back of his bike.   “I thought it would be kind of cool to have her riding with me,” he said.

EVENT INFORMATION:
The inaugural Shakes, Rattles, Rides and Rods fundraiser to benefit foundations working toward treatments and a cure for Parkinson’s disease will be held from noon to 6 p.m. on Saturday, Feb. 21, at the Hidden River Travel Resort, at 12500 McMullen Loop in Riverview.

The event, hosted by AMVETS Post 44, American Legion Riders Alafia Post 148, and Go For A Ride Magazine, will first kick off with a motorcycle ride. Riders should register at the American Legion Alafia Post 148 at 7240 U.S. 301 in Riverview between 10 a.m. and 11:45 a.m.

Requested donations to the event are $15 per person or $25 per couple. It includes food, entertainment, music, dancing, a live auction, vendors, and a grand-prize drawing.

All proceeds go to the Frances J. Zesiewicz Center for Parkinson’s Disease at USF, as well as the Michael J. Fox Foundation.

For more information, call Rick Karczewski at (813) 810-5001.

News release by Saundra Amrhein

Media contact: Anne DeLotto Baier, USF Health Communications
abaier@health.usf.edu or (813) 974-3303

Two USF Health students were major contributing authors to a new book providing a comprehensive description of current treatment options for Parkinson’s disease.

Madeline Snyder, a third-year medical student at the Morsani College of Medicine, and Michael Carranza, who recently graduated from MCOM with a M.S. degree in neuropharmacology and is now a student at Nova Southeastern University College of Osteopathic Medicine, helped write Parkinson’s Disease: A Guide to Medical Treatment  published in May by SEEd.

“It’s very unusual for students to produce a book this specialized.  That says a lot about their commitment and dedication to Neurology and Parkinson’s disease patients,” said neurology professor Theresa Zesiewicz, MD, director of the Frances J. Zesiewicz Center and Foundation for Parkinson’s Disease at USF, named in memory of her mother who suffered from the disease.

 
USF medical student Madeline Snyder was one of the book’s student authors

The book’s other authors were Dr. Zesiewicz and Jessica Davenport Shaw, MPH, a research support specialist in the USF Department of Neurology and a 2011 graduate of the USF College of Public Health specializing in epidemiology.

Snyder, 25, was not new to publishing. As a USF medical student and an undergraduate majoring in biology at the University of Pennsylvania, she had already authored several papers on neurological topics in peer-reviewed journals.

But those experiences weren’t as all-encompassing as producing the book, which required extensive literature reviews and other secondary research, documentation, collaboration among the authors, writing and re-writing, massive editing and re-editing.

“I read hundreds of papers,” said Snyder, who tackled the project between the first and second year of medical school as part of her ongoing scholarly concentration program in research. “The whole process gave me a lot of hope for patients, because I became aware of all the studies done over the past decades and advances in treatment being made.”

USF neurology professor Dr. Theresa Zesiewicz, right, with her mother. The Frances J. Zesiewicz Center and Foundation for Parkinson’s Disease at USF, is named in honor of Dr. Zesiwicz’ mother, who suffered from the movement disorder.

The book is intended as a resource for a wide audience, including patients and caregivers as well as neurologists and primary care physicians.  Its eight chapters cover everything from diagnosis and pathophysiology to the pharmacology of anti-Parkinson’s drugs and medical treatment of motor and non-motor symptoms. 

The book’s final chapter – a question-and-answer section addressing common patient concerns – was an idea Snyder developed with the help of Dr. Zesiewicz.  Snyder says she kept note of the questions frequently asked by patients while she was in the clinic shadowing Dr. Zesiewicz, including inquiries about the potential benefit of complementary therapies like Tai Chi and yoga.

Snyder also leads a pilot study at USF investigating whether subtle changes in handwriting may be an early biomarker for Parkinson’s disease – one that may help detect those at risk for the progressive movement disorder before symptoms of tremor, rigidity and imbalance appear.

Snyder credited Dr. Zesiewicz — known as “Dr. Z” by students, colleagues and patients — with being a role model who encourages research and other scholarly pursuits seeking to improve patient care and quality of life.

“Dr. Z was definitely our guide in the adventure of putting together this book,” she said. “She brings light and energy to everything she does… She spends a lot of time with each of her patients, and they all smile when she enters the room. That’s the kind of physician I want to be.”