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Patients have always been the center of the yearly scientific symposium hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the University of South Florida Ataxia Research Center.

But, for the 8^th annual symposium held Sept. 15 at USF’s Gibbons Alumni Center, patients took on an even more prominent role. The panel discussion in which they share their stories about living with the rare, but devastating, progressive neurodegenerative disease, including patient participation in clinical trials, was moved up in the program format.

The 8th annual scientific symposium hosted by FARA and USF was held in the USF Gibbons Alumni Center.  More than 750 in the FA community listened to the latest perspectives on ataxia research from patients and scientists, in person and internationally via live-stream.

And this year, led by FARA spokesperson Kyle Bryant as moderator, the four patient panelists were the ones driving the conversation with leading researchers from academia and industry who sat onstage beside these young adults to discuss the latest advances in the search for effective treatments and, ultimately, a cure.

More than 250 attendees gathered at the USF Gibbons Alumni Center for the symposium, which was also live-streamed and viewed worldwide by those in the FA community, over 500 people in eight countries. The symposium “Understanding Energy for a Cure” kicked off a series of events in Tampa Bay to raise awareness about FA, culminating Sept. 17 with the FARA Energy Ball gala, which this year raised $2 million to benefit innovative ataxia research.

The patient panel, moderated by FARA spokesperson Kyle Bryant (far left), helped drive the conversation with leading researchers. Participants were, from left, Alex Fielding, Sean Baumstark, Alison Avery and Anna Gordon.

“My parents and sister never really let me believe that Friedreich’s ataxia was going to stop me,” said panelist Alison Avery, 22, diagnosed with Friedreich’s ataxia at age 18, who is interning with the National Football League’s social responsibility department in NYC following college graduation. “It may have changed the way that I do certain things, but right now I’m living on my own in New York City, and that’s something not everyone would do, whether or not they have FA.”

Alison participates in the “Cardiac MRI and Biomarkers in Friedreich’s Ataxia” study at Children’s Hospital of Philadelphia and another evaluating the relationship between exercise performance and neurological/cardiac status and overall functioning in children and adults with FA. “I’m excited to be able to share my perspective on being involved in different research studies,” she said. “I feel like that’s something more people should know, especially the researchers — about how patients actually feel about trials and studies.”

USF System President Judy Genshaft said USF has made neurosciences, including ataxia research, a high institutional research priority.

Friedreich’s ataxia typically strikes in childhood or adolescence and leads to a progressive loss of coordination and muscle strength, eventually robbing young people of their energy and ability to walk. While the neurological symptoms are most visible, FA is a multisystem disease that can adversely affect cardiac function, metabolism, vision, hearing and the skeletal system. There is currently no approved treatment for FA.

“Throughout the history of this event, the one constant has been how incredibly motivating and inspiring it is to hear from patients and their families who never fail to share one valuable message: ‘Live life to its fullest despite the challenges of Friedreich’s ataxia,’” said USF System President Judy Genshaft in her symposium welcome remarks.

FARA Executive Director Jennifer Farmer introduced the patients and provided insights on their participation in studies and clinical trials.

The USF Health Morsani College of Medicine is one of 10 sites in the international FARA Collaborative Clinical Research Network, all working to discover treatments that can attack FA on different fronts and improve the quality of life for patients.

“We’ve made this a high research priority within the institution,” President Genshaft said. “Over the last 20 years FARA’s international collaborative of researchers has increased the pace in the fight against FA. Today more than 20 drugs are in the treatment pipeline and ongoing studies are working toward the discovery of new therapies… We have every reason to be hopeful, but we do know there is more work to be done.”

Theresa Zesiewicz, MD, professor of neurology and director of the USF Ataxia Research Center, presented promising preliminary results from two clinical trials conducted at USF, among other sites. – Photo by Kent Ross

Theresa Zesiewicz, MD, professor of neurology and director of the USF Ataxia Research Center, updated attendees on the center’s initiatives.

“We started out at USF with one clinical trial eight years ago, and now we have five or six clinical trials and each (investigational) drug works differently,” Dr. Zesiewicz said. “Some drugs work to increase frataxin (the protein depleted in those with FA), some drugs work on inflammation, some work as strong antioxidants. So, there may not be one magic bullet to stop this disease; rather, it may require a cocktail of therapies, a conglomerate of different compounds to help delay or stop the disease process.”

David Lynch, MD, PhD, (center) lead investigator for the FA Natural History Study at Children’s Hospital of Philadelphia, responds to a patient question. He was joined in the discussion of clinical trials by Martin Delatycki, PhD, (far left) of Murdoch Children’s Research Institute, Melbourne, Australia.

Some promising preliminary results for two clinical trials conducted at USF, among other sites, were announced by lead investigator Dr. Zesiewicz. Both studies were done in collaboration with FARA.

• EPI-743 Safety and Effectiveness Study: The Phase 2 open-label extension study, sponsored by Edison Pharmaceuticals, tested the effectiveness of the potent antioxidant EPI 743 primarily on vision, and secondarily, on neurological function in adult patients with FA. After two years of study and a year of data analysis, the researchers found that patients taking EPI-743 from the study’s start demonstrated markedly less disease progression than would be expected in the natural history of the disease. The improvement in neurological function was dose-dependent, and although the last 18 months of the study were open-label, patients and investigators were blinded to the drug dose allocation. Additional studies of EPI-743 are planned in pediatric patients and those with point mutations.

FARA President Ron Bartek thanked everyone in the room, including researchers, pharmaceutical partners and patients and their families, for working together to advance discoveries to “slow, stop and reverse” Friedreich’s ataxia.

• Retrotope RT001 Phase 1/2: The randomized double-blind, placebo-controlled trial evaluated the safety, tolerability and early effectiveness of the stabilized fatty acid RT001 in adult patients with FA. In the small, 28-day study, researchers found that the drug was safe, well tolerated at high doses and rapidly absorbed to target levels, with early signs of effectiveness. Earlier this year, the FDA granted Retrotope orphan drug designation for RT001 in FA.

The scientist and physician panelists at the symposium covered four areas of FA research:

• Basic and Discovery Science: Helene Puccio, PhD; Marek Napierala, PhD; and Jordi Magrane, PhD
• Drug Development and Advancing Treatments: Mark Payne, MD; and Barry Byrne, MD, PhD.
• FA Biomarkers: Kimberly Lee Lin, MD; Angel Martin, PhD student; and Christophe Lenglet, PhD.
• Clinical Trials and Translating Treatments to Improved Care: Martin Delatycki, PhD; and David Lynch, MD, PhD.

The researchers discussed their scholarly work, progress beyond their laboratories and its relevance to advancing treatments. They also emphasized their passion for FA science and personal commitment to patients.

Scientists participating in the Basic and Discovery Science panel discussion were, from left, Jordi Magrane, PhD, of the Brain and Mind Research Institute, Weill Cornell Medical College; Marek Napierala, PhD, of the University of Alabama; and Helene Puccio, PhD, of the Institute of Genetics and Molecular and Cellular Biology, University of Strasbourg.

Moving from treating symptoms to slowing and stopping progression to reversing disease is “not an overnight event,” said David Lynch, MD, PhD, lead investigator for the FARA Natural History Study at Children’s Hospital of Philadelphia. “So, in 15 years we may look back and talk not about the advance but about the 15 advances from each of 15 clinical trials superimposed on top of one another, eventually leading to that four letter word — cure.”

Answering patient questions on drug development and advancing treatments were physician-scientists Barry Byrne, MD, PhD, (left) of the University of Florida College of Medicine; and R. Mark Payne, MD, of Indiana University School of Medicine.

Despite the challenges, the researchers agreed that the steadfast determination and resilience of patients and their families energizes them to keep working toward a cure.

“Everything we do is for the patients, and we are all in this together trying to find a treatment and cure for Friedreich’s ataxia,” said USF’s Dr. Zesiewicz. “That’s the only reason we’re here.”

Participants in the FA BioMarkers panel discussion were, from left, Kimberly Lee Lin, MD, of Children’s Hospital of Philadelphia; Christophe Lenglet, PhD, of the Institute for Translational Neuroscience, University of Minnesota; and Angel Martin, a PhD candidate at Duke University.

Alison Avery, second from right, credits her family — sister Laurel Avery (left) and parents Paul and Suzanne Avery — with “never really letting me believe that Friedreich’s ataxia is going to stop me.”

Dr. Zesiewicz with members of the USF Ataxia Research Center, one of 10 sites in the international FARA Collaborative Clinical Research Network. – Photo by Kent Ross

Photos by Eric Younghans and video by Sandra C. Roa, USF Health Communications and Marketing

Bridging laboratory and clinical sciences, the study aims to improve the health outcomes  of pregnancies complicated by poor fetal growth

Tampa, FL (Sept. 28, 2015) – The USF Health Morsani College of Medicine has received a $4-million National Institutes of Health grant that will employ new imaging technologies and test biomarkers in the blood to determine whether abnormalities in the smallest blood vessels of the placenta and negative environmental influences, particularly smoking, cause fetal growth restriction (FGR).

The ultimate goal of the four-year study is to design a reliable way to predict poor growth of the fetus earlier in pregnancy so that physicians can intervene sooner to help prevent stillbirth, Cesarean delivery, decreased oxygen levels and other adverse outcomes.

The USF research award (1U01HD087213-01) was announced today as one of 19 projects funded by the Human Placenta Project — an initiative launched by the NIH’s Eunice Kennedy Shriver National Institute of Child Health and Development to better understand the role of the placenta in health and disease.

Anthony Odibo, MD (left) and Umit Kayisli, PhD, of the USF Health Department of Obstetrics and Gynecology, are co-principal investigators of a $4-million Human Placenta Project — one of 19 new projects awarded in the U.S. and Canada by the NIH.

“I am so proud of our team,” said Charles J. Lockwood, MD,  dean of the USF Health Morsani College of Medicine and senior vice president for USF Health. “This is an important NIH initiative which addresses the common source of most major adverse pregnancy events – abnormal placentation.”

“In the past, it has been challenging to identify which women may benefit (from early therapeutic intervention), because they are at high risk for fetal growth restriction,” said co-principal investigator Anthony Odibo, MD, professor in the USF Department of Obstetrics and Gynecology.  “But powerful new imaging technology gives us the opportunity to really visualize all the blood vessels in the placenta, study the underlying problem of growth restriction, and create a highly predictive model for identifying small babies at risk of FGR.”

The USF grant, bridging laboratory and clinical sciences, will be led by Dr. Odibo and co-principal investigator Umit Kayisli, PhD, associate professor of obstetrics and gynecology.  Dr. Odibo, specializing in maternal-fetal medicine, is an expert in fetal therapy and directs the USF Fetal Care Center at Tampa General Hospital.  Dr. Kayisli specializes in molecular and cellular biology in reproduction and its clinical implications.

They will work on the NIH project with USF Ob/Gyn co-investigators Charles J. Lockwood, MD, Frederick Schatz, PhD, and Ozlem Guzeloglu-Kayisli, PhD, and with Rajendra Kedar, MD, from the USF Department of Radiology.  USF colleagues at Necker Hospital in Paris and at Oakland University William Beaumont School of Medicine in Rochester, MI, will also collaborate.

Fetal growth restriction (FGR), affecting up to 10 percent of all pregnancies, is commonly defined as fetal weight below the 10^th percentile for gestational age as determined by ultrasound. The condition remains a leading contributor worldwide to the death and illness of babies before and after birth.

Placental function – the ability of the critical organ to shuttle blood, oxygen and nutrients from mother to fetus through an intricate network of blood vessels – is inadequate in pregnancies complicated by FGR.  But predicting FGR has been difficult, because until recently imaging technologies have not been sensitive nor specific enough to clearly detect the smallest blood vessels in the placenta and monitor the flow of blood through this branching microvasculature.

Dr. Odibo points to an ultrasound image of the placenta, a critical organ that shuttles blood, oxygen and nutrients from mother to fetus through an intricate network of blood vessels.

For the USF study, researchers will use two of the latest technologies – superb microvascular imaging, or SMI ultrasound, and blood oxygen level-dependent magnetic resonance imaging, or BOLD MRI.

The investigators will compare biopsies of placenta from normal and FGR-complicated pregnancies in the laboratory and correlate them with the imaging assessments of the placental microvasculature.  They will also study how smoking affects the microvasculature and the potential link with FGR.

“The results obtained from SMI ultrasound and BOLD MRI combined with changes in expression levels of several biomarkers and epigenetic modifications in response to smoking will be instrumental in developing a predictive model for pregnancies at high risk for fetal growth restriction and improving the sensitivity and specificity of a potential early diagnosis and treatment of FGR,” Dr. Kayisli said.

For a list of all new grants awarded as part of the NIH Human Placenta Project, go to http://www.nichd.nih.gov/news/releases/Pages/092815-NIH-awards-HPP.aspx.

-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 Physician’s Group. The University of South Florida is a Top 50 research university in total research expenditures among both public and private institutions nationwide, according to the National Science Foundation.  For more information, visit www.health.usf.edu

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

Photos by Eric Younghans, USF Health Communications and Marketing

The NIA-funded study focuses on those who care for people with dementia and Alzheimer’s disease at home

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Tampa, FL (July 9, 2013) – The University of South Florida College of Nursing is conducting research to improve sleep in those caring for people with dementia and Alzheimer’s disease, with the aim of determining if better sleep affects heart health. The $1.9-million, four-year study funded by National Institute on Aging (NIA), “Improving Dementia Caregiver Sleep and the Effect on Heart Disease Biomarkers,” is led by USF College of Nursing Professor and Endowed Chair Meredeth Rowe, RN, PhD, FGSA, FAAN.

Fifteen million Americans provided an estimated 17.5 billion hours of unpaid care to people with Alzheimer’s disease and other dementias in 2012, according to the Alzheimer’s Association. Recent studies indicate that caregivers, unpaid family members or friends who assist people with Alzheimer’s with daily activities, may experience sleep loss from the constant demands of caregiving.

The College of Nursing study tests the relationship between caregiver sleep and heart disease to help understand what negatively affects caregiver health.

USF College of Nursing’s Dr. Meredeth Rowe places one of the monitoring device’s sensors beneath the mattress. The sensor detects movement and sounds an alarm to alert the caregiver if the person with Alzheimer’s disease starts to get out of bed.

“Caregiving and lack of sleep each separately increase the risk of heart disease,” Dr. Rowe said. “We want to discover whether improving sleep in caregivers lowers that risk.”

According to the 2006 Institute of Medicine (IOM) report “Sleep Disorders and Sleep Deprivation: An Unmet Public Health Problem,” the cumulative effects of sleep loss and sleep disorders have been associated with a wide range of adverse health consequences including increased risk of hypertension, diabetes, obesity, depression, heart attack, and stroke.

“The College of Nursing at the University of South Florida is transforming lives through research to improve the health of patients and their caregivers,” said Dianne Morrison-Beedy, PhD, RN, WHNP-BC, FNAP, FAANP, FAAN, senior associate vice president of USF Health and dean of the College of Nursing. “We’re making life better by transforming the health care of the family members who provide essential home care for persons with dementia. These caregivers fill an often unrecognized role in the healthcare system.”

The primary focus of Dr. Rowe’s research is finding an effective and easy-to-use treatment that can improve sleep in Alzheimer’s caregivers to increase their overall health and reduce the nursing home placement of people with dementia.

“When I was working on ways to provide caregivers better quality of sleep, I looked for technologies that might help and there were none,” Dr. Rowe said. “I realized that unless I invented a system that allowed them to sleep well, I was never going to have an impact on improving their sleep.”

Dr. Rowe explains to a caregiver how the integrated nighttime monitoring system tracks the bed occupancy and movement patterns around the house of a loved one with dementia. The goal is to allow the caregiver to rest easier through the night while maintaining the safety of the person with dementia.

A number of sensors can be placed around the house, including doorways, to analyze movement patterns and provide customized, specific alerts.

CareAlert, a new night monitoring system designed by Dr. Rowe and Caregiver Watch, LLC, provides reliable alerts to caregivers whenever a person with dementia leaves the bed and wanders through the house. The system helps the caregiver rest easier through the night, and improves the overall safety of the person with dementia. The findings of a study testing the effectiveness of CareAlert were published by Dr. Rowe and colleagues in Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association, in 2009. The researchers reported that use of CareAlert reduced nighttime injuries and unattended home exits by 85 percent over a period of 12 months.

“Dr. Rowe’s research seeks not only to improve caregiver sleep, but also to better understand the relationships between sleep and changes in heart health,” said Cindy L. Munro, PhD, RN, ANP-BC, FAAN, professor and associate dean for research and innovation at the USF College of Nursing.

In the latest research, Dr. Rowe is evaluating whether a combined intervention using  CareAlert and cognitive-behavioral therapy for insomnia improves sleep in individuals who care for people with dementia and Alzheimer’s who wake up at night. The study, conducted in the USF College of Nursing Caregiving Laboratory, will include as many as 100 participants. All will receive the CareAlert device and one of two sleep therapies assigned at random.

Dr. Rowe, a national expert in caregiver-related issues, worked with a company to design an evidence-based monitoring system with technology responsive to caregiver needs.

Rita F. D’Aoust, PhD, ACNP, ANP-BC, CNE, FAANP, FNAP, associate professor and associate dean of academic affairs and interprofessional initiatives at the USF College of Nursing, and Maureen E. Groer, RN, PhD, FAAN, Gordon Keller professor, are study co-investigators. Dr. D’Aoust studies the associations between vigilance and sympathetic nervous system activity, and Dr. Groer leads the design and analysis of biologic markers for heart disease. Others contributing to this research project include Brandi Mallek, research project manager; Taujihana Brown, research assistant; Glenna Brewster, predoctoral fellow; Milora Morley, undergraduate public health honors student.

The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency and the leading supporter of biomedical research in the world. NIA, one of the 27 institutes and centers at NIH, helps understand the nature of aging and supports the health and well-being of older adults. Dr. Rowe has received many NIH grants throughout her research career to study people with Alzheimer’s disease or dementia and their caregivers. The USF College of Nursing ranks first in Florida and 24th in the nation in NIH funding, for both public and private schools of nursing.

Dr. Maureen Groer, a co-investigator for the caregiver sleep and heart disease risk study, watches as research lab technician Nicole Williams drops processed blood into a machine that will identify biomarkers of heart disease.

Dr. Rowe is currently enrolling participants for this study. For more information, contact USF College of Nursing Research Project Manager Brandi Mallek, at (813) 974-1827 or bmallek@health.usf.edu.

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 Biomedical Sciences and the School of Physical Therapy and Rehabilitation Sciences; and the USF Physician’s Group. The University of South Florida is a global research university ranked 50^th in the nation by the National Science Foundation for both federal and total research expenditures among all U.S. universities. For more information, visit www.health.usf.edu.

Media contact:
Ashlea Bailey, College of Nursing Communications 
(813)396-9642 or  ahudak@health.usf.edu

All photos with Dr. Rowe taken at the USF Health Byrd Alzheimer’s Institute’s Center for Memory C.A.R.E  by Eric Younghans, USF Health Communications
Video by Andy Faza, USF College of Nursing Communications & Marketing