USF Health preclinical study tests several formulations of chitosan-enriched siRNA nanoparticles intended to improve gene therapy targeting neurodegenerative diseases

Neurologist Juan Sanchez-Ramos, MD, PhD, director of USF Health’s HDSA Huntington’s Disease Center of Excellence, examines patient and clinical trial participant Brittany Bosson.

Huntington’s disease (HD) is a hereditary brain disease that typically strikes adults in the prime of life – leading to progressive deterioration of movement, mood and thinking. While some drugs temporarily alleviate symptoms, currently no therapies prevent, slow or stop the course of HD.

Juan Sanchez-Ramos, MD, PhD, the Helen Ellis Professor of Neurology and director of  the HDSA Huntington’s Disease Center of Excellence, University of South Florida Health (USF Health), sees firsthand how this devastating illness – sometimes described as a mix of Parkinson’s disease, ALS and Alzheimer’s disease — affects patients and their families.  For the last several years, even as he leads clinical trials evaluating potential new drugs, the physician-scientist has worked with a mouse model of HD to develop and test a nanoparticle system that can precisely deliver gene therapy from the nose to areas of the brain most affected by HD.

He is closer than ever before to a viable noninvasive treatment – one that could be administered by nasal spray or drops, rather than spinal puncture or direct injection into the brain.

In a preclinical study published Oct. 27 in Nanomedicine: Nanotechnology, Biology and Medicine, senior author Dr. Sanchez-Ramos and colleagues build on their earlier findings demonstrating that chitosan-enriched, manganese-coated nanoparticles loaded with small interfering RNA (siRNA) could be successfully delivered by nose drops to targeted parts of the brain affected by HD.  In a Huntington’s disease mouse model the nanoparticles reduced expression of the mutated HTT gene that causes HD by at least 50% in four regions: the olfactory bulb, striatum, hippocampus and cortex. The defective HTT gene leads to production of a toxic form of protein, known as the huntingtin protein. In essence, this new treatment silences the genetic message “telling” a cell to generate more huntingtin proteins. To ultimately benefit patients, the abnormal protein production must be reduced enough to block or slow the dysfunction and eventual loss of nerve cells accounting for clinical symptoms.

“Our nose-to-brain approach for delivery of gene therapies is non-invasive, safe and effective,” said Dr. Sanchez-Ramos, a co-inventor of the novel anti-HTT siRNA nanoparticle delivery system patented by USF.

Searching for ways to optimize HD gene silencing

For the latest study, reported in Nanomedicine, Dr. Sanchez-Ramos collaborated with researchers from the USF Health Department of Neurology and the University of Massachusetts Medical School’s RNA Therapeutics Institute.  Seeking to optimize HD gene silencing when the siRNA is delivered by a nasal route, the team tested different formulations and sizes of the nanoparticles in a mouse model expressing the human HD gene. Among their findings:

— Four different versions of the nanoparticles tested lowered HD gene expression in the brain by 50%. However, lowering levels of the toxic huntingtin protein in brain tissue took longer, with the highest reduction of the protein (53%) seen in the olfactory bulb at the base of the brain and the lowest (38%) in the cerebral cortex, the brain’s outer layer. Also, simply administering “naked” siRNA through the nose (without the protective chitosan encasement) did little to reduce HD gene expression even though previous research has shown similar naked siRNA injected directly into the brain was highly effective.

— Enclosing the siRNA in chitosan protected the silencing RNA from being prematurely degraded “en route” to its HD brain targets. The compound chitosan is derived from the hard outer skeleton of shellfish or the external skeleton of insects. Encapsulating siRNA into a chitosan nanoparticle allowed the silencing RNA to be enriched to higher doses without damaging the molecule, resulting in significant reduction in HD gene expression, the researchers report.

— Increasing the number siRNA nanoparticles within a defined dose of nose drops is a key to improving therapeutic potential. “The ability to fabricate concentrated NP (nanoparticle) preparations without damaging siRNA content is a critical factor for successful intranasal delivery of gene silencing agents,” the researchers concluded.

A major challenge of gene therapy for HD and other neurodegenerative diseases has been getting the molecules intended to replace a missing gene or suppress an overactive gene past the blood-brain barrier, a kind of defensive wall that selectively filters which molecules can enter the brain from circulating blood.

But over the last several years, research progressed in overcoming this barrier and promising laboratory findings set the stage for clinical trials in patients with HD.

For example, led by Dr. Sanchez-Ramos, USF Health is the only Florida site participating in the Roche-sponsored GENERATION HD1 Study. This pivotal phase 3 international clinical trial is testing whether a huntingtin-lowering, antisense oligonucleotide drug can halt underlying pathology of the disease enough to improve symptoms in adult patients. The injectable drug, administered directly into the cerebral-spinal fluid, successfully bypasses the blood-brain barrier and stopped disease progression in laboratory models. However, the investigational drug must be administered every two months by lumbar puncture at the clinic.

The normal huntingtin gene contains a DNA alphabet that repeats the letters C-A-G as many as 26 times, but people who develop Huntington’s disease have an excessive number of these consecutive C-A-G triplet repeats — greater than 39.| Graphic by Sandra C. Roa

Working toward a simpler, noninvasive treatment

With a chronic illness that gradually encompasses the entire central nervous system, like HD, even minimally-invasive injections with fine needles or infusions may pose risks of infection or other complications associated with neurosurgical procedures, Dr. Sanchez-Ramos said. So, he continues to work toward a noninvasive nose-to-brain treatment that would be simpler to repeat and well-tolerated by patients over their lifetime.

Dr. Sanchez-Ramos says the idea for incorporating nontoxic amounts of manganese chelate into the chitosan-based nanoparticles to help gene therapy delivery was sparked by early studies investigating how welders exposed to high levels of neurotoxic manganese oxide from welding fumes developed Parkinson’s disease symptoms.  It turns out that the olfactory nerve has an affinity for the chemical manganese.

“Manganese is good at guiding our nanoparticles from the nasal passages to the olfactory nerves and transporting the particles directly to structures deep in the brain… Realizing that was one of our biggest breakthroughs,” he said.  Manganese also permits the nanoparticles to be visualized by MRI imaging, so that their distribution and accumulation in different regions of brain can be tracked.

The nose-to-brain method of delivering the manganese-containing siRNA nanoparticles needs to be tested in a larger-brain animal model before moving to human trials.

The USF Health study was supported by a grant from the National Institute of Health’s National Institute of Neurological Disorders and Stroke.

As his preclinical research on nose-to-brain delivery of gene therapy for Huntington’s disease progresses, Dr. Sanchez-Ramos serves as Florida principal investigator for a worldwide clinical trial testing an injectable drug designed to slow the progression of Huntington’s disease.

-Photos by Allison Long, USF Health Communications and Marketing

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USF Health has established a Neurocardiogenetics Clinic focused on improving the quality of life for patients with hereditary neuromuscular disorders who are at risk for or already experiencing cardiac complications. Innovative genetic research is a central component of the new clinic.

The multidisciplinary clinic teams faculty and staff with clinical expertise in cardiology, genetics and neurology. The clinic is directed by Aarti Patel, MD, assistant professor of cardiology and director of the Cardiac Imaging Fellowship Program at USF Health in collaboration with Thomas McDonald, MD, professor of cardiology and molecular pharmacology and physiology, and a member of the USF Health Heart Institute, and Theresa Zesiewicz, MD, professor of neurology and director of the USF Health Ataxia Research Center. Patients receive a complete cardiac examination and testing along with a comprehensive family genetic evaluation from providers familiar with their neurological history.

“Combining all that information, we are able to come up with a personalized treatment plan,” Dr. Patel said.

Many patients initially seen at the Neurocardiogenetics Clinic have been diagnosed with Friedreich’s ataxia (FA), a rare, debilitating and life-shortening neuromuscular disorder that usually strikes in childhood. At some point in their lives 55 to 60-percent of FA patients develop some form of cardiac disease. The most devastating of these is cardiomyopathy, a heart muscle disease that limits blood pumped to the rest of the body and increases risk for heart failure and abnormal heart rhythms.

“FA is a multisystem disease that affects many different parts of the body, including the heart,” Dr. Zesiewicz said. “And one of its most sinister complications is cardiomyopathy, an enlarged heart.”

The Neurocardiogenetic clinic offers patients and their families the opportunity to participate in ongoing genetic studies exploring potential links between heart disease and neuromuscular diseases like FA and certain types of other ataxias and muscular dystrophies.

Dr. Aarti Patel (left), assistant professor of cardiology, collaborates with Dr. Thomas McDonald, professor of molecular pharmacology and physiology, at the new USF Health Neurocardiogenetics Clinic. Dr. Theresa Zesiewicz. professor of neurology (not pictured), is another collaborator.

While a deficiency in the frataxin protein can lead to fibrosis and scarring of heart muscle tissue in FA, competing theories exist about how a genetic mutation may actually result in heart problems in this particular ataxia and other neuromuscular disorders.

Dr. McDonald’s research team is searching for answers to many questions, including how different genetic variations may lead to cardiac abnormalities and affect the severity of heart disease.

Small blood samples are collected from patient volunteers with different inherited neuromuscular and/or cardiac disorders, as well as family members who are unaffected genetic carriers of neuromuscular diseases. In laboratory cell culture dishes, USF Health researchers genetically reprogram the blood cells into pluripotent stem cells that can grow into any cell type. Then they induce these stem cells — containing the same genetic make-up as the patient who provided blood — to become nerve cells and heart muscle cells.

The goal is to work out at a molecular level how FA or other inherited neuromuscular diseases damage the heart muscle. Once that is achieved, Dr. McDonald said, the “disease in a dish” can be used to identify potential drug targets and test treatment options on the patient’s own cells.

“With stem cell technology and disease-in-a-dish modeling, we’re looking for the earliest changes in either a heart or a nerve cell (that lead to disease),” he said. “A big unanswered question is whether the molecular or cellular process happening in the nerve is the same as that damaging the heart muscle. By using stem cell-based research we hope to be able to determine if there is a connection between the nerve and the heart – or whether it’s a separate, independent process but based upon the same genetic defect.”

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The collaborative research builds upon robust data collected by doctors in the clinic, including the wide range of symptoms seen in patients with hereditary neurological disorders involving cardiac complications.

Ultimately, that can help improve understanding of why genetic variations in certain neuromuscular diseases cause cardiac complications, Dr. McDonald said. “Having a clear clinical picture of each research participant will help us correlate the genetics and function of cells we see in the laboratory with how that translates to disease progression in the individual.”

“With careful clinical surveillance and novel biomarkers, our hope is that we will be able to predict which patients and their family members are at highest risk for cardiovascular disease so we can intervene early with effective treatment,” Dr. Patel said. “We’re hoping to identify patients even before they develop cardiac symptoms, so we can prevent heart disease.”

-Video and photos by Allison Long, USF Health Communications and Marketing

Clifton (Cliff) Gooch, MD, professor and chair of the USF Health Morsani College of Medicine’s Department of Neurology, was recently appointed to the national ALS Association Board of Trustees.  He is one of four new members of the 25-member national board.

The Association is the world’s largest private funder of research for amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord.

A leader in the ALS community, Dr. Gooch developed his interest in ALS at the beginning of his neurology training at Baylor University College of Medicine in Houston, Texas.  He has engaged in ALS research, clinical care, and administration throughout his career.

He began his career as a junior faculty member with Baylor’s ALS Center in the early 1990s, then moved to Columbia University in New York, NY, in 1999, where he was part of the Eleanor and Lou Gehrig ALS Center and the Columbia Motor Neuron Research Center. Since 2008, he has led the Department of Neurology at the University of South Florida in Tampa, Fla. The university has one of the largest ALS clinical care and clinical research centers in the Southeast.

Dr. Gooch has held numerous leadership positions at USF, including establishing and directing the USF Neuroscience Collaborative for interdisciplinary research and founding the USF ALS Center. During his tenure, the Neurology Department more than tripled in size, greatly expanded its research portfolio, and now conducts more than 100 clinical trials a year.

In addition to his national leadership position as president-elect of the Association of University Professors of Neurology, Dr. Gooch serves as treasurer of the American Neurological Association (ANA) Board of Directors. He is a fellow of both the American Academy of Neurology and the ANA, and highly sought as a speaker by many organizations across the country and internationally.

USF Health doctors evaluate for refractory epilepsy, experts in treating surgical candidates

Some people pay it forward by donating to a charity or working in a soup kitchen. Six years after her own epilepsy surgery, Letitia Browne-James found an even better way to thank USF Health neurologist and epileptologist Selim Benbadis, MD, and USF Health neurosurgeon Fernando Vale, MD. Browne-James volunteers as a patient advocate.

Singing the praises of Dr. Benbadis and Dr. Vale, Browne-James said the two were a couple of the most compassionate and skilled physicians she has ever worked with. “And, as a way to pay it forward, I wanted to help patients,” she said.

Letitia Browne-James

“I’ve used my education in forms of advocacy for mental health and social justice. I also use it in the medical field by trying to advocate for patients, and teaching them how to get educated and how to self-advocate,” the St. Thomas, U.S. Virgin Islands native said.  “Four patients who suffered from epilepsy – either because they heard my story, or I talked to them and helped them get to the right place — are now seizure free.”

“The delay for patients like Letitia to have epilepsy surgery is more than 15 years, so it is very important to have patient advocates speak up and educate patients about exploring options other than multiple medications,” said Dr. Benbadis, a professor and director of the Comprehensive Epilepsy Program at USF Health and Tampa General Hospital.

Suffering from epileptic seizures since she was a baby, Browne-James was diagnosed officially with epilepsy at age 12.  She suffered seizures throughout high school, college, graduate school and beyond.  After her 27th birthday, the seizures became increasingly violent.  The news at the doctor’s office wasn’t much better.

Browne-James suffered from epileptic seizures since she was a baby. She is pictured here at age 5.

“My doctor at the time said there wasn’t anything we could do, other than switching up the medication frequently. But that action didn’t help,” Browne-James said.

A licensed mental health counselor in Florida, Browne-James found a top-notch psychiatrist to see if she was having pseudo-symptoms, possibly pseudo-seizures. After a psychological assessment, the psychiatrist ruled out pseudo-seizures.  She instead was diagnosed with anxiety due to the seizure disorder and prescribed Xanax.

She had enough and began extensively researching the subject online.  Her Google searches led her to a new kind of a doctor: an epileptologist, a neurologist who specializes in epilepsy that’s hard to diagnose and treat, or refractory.

After an initial meeting with epileptologist Dr. Ahmed Sadek of Neurological Services of Orlando, Browne-James was diagnosed with refractory epilepsy.   

Dr. Sadek explained to Browne-James why years of tests had failed to show seizure activity in her brain. He put her through a different series of tests over a five day period on an epilepsy monitoring unit.  His conclusion: she was a perfect candidate for epilepsy surgery.

Browne-James, right, at her graduation from Charlotte Amalie High School in 1999 (in St. Thomas US Virgin Islands), with friend Yonette Francis.

During a phone consultation with physicians at Mayo Clinic in Jacksonville, they recommended that she contact Dr. Benbadis and Dr. Vale of USF Health because Dr. Vale was an expert in epilepsy surgery and performed it often.

“I thought ‘if the Mayo Clinic says these doctors are good, then they are for me,”’ Browne-James said.

After her initial visits with Dr. Benbadis, she underwent a series of medical tests.  USF Health neuropsychologist Michael Schoenberg, PhD, performed IQ tests to determine if Browne-James would maintain normal cognitive functioning after surgery. These tests were normal and also helped further determine she was an ideal candidate for the surgery.

She was ready to start a fresh chapter of her life.

Browne-James with husband Jonah James, Jr., at their wedding in 2008.

“I had no anxiety whatsoever about having brain surgery, because I knew the way my life was deteriorating from epilepsy could not continue. It would kill me, whether physically or from the emotional stress. I am very grateful to them,” Browne-James said.

Dr. Vale performed the surgery in August 2012, and Browne-James has been seizure free since.  In IQ tests following the surgery, Browne-James scored the same in some areas, but in some areas there were definite improvements.

This summer she will finish her doctorate online at Walden University in counselor education and supervision with a specialization in counseling and social change.  “Reading and comprehension come easier,” Browne-James said.  “My brain feels free, not clouded like it was before the surgery.”

“The reason her mind is clearer is two-fold,” Dr. Benbadis said. “No more seizures, and fewer or no medications. Seizure medications work, but often cause side effects like fatigue, dizziness and mental fogginess.”

An adjunct professor at Stetson University, Browne-James hopes her doctorate will open more doors to further research and teaching.

“My work in the medical field is not exclusive to epilepsy patients,” she said. “I want to get all patients to understand the importance of self-advocacy and being an informed patient.  It’s important to understand your doctor works for you.”

Roni-Kay Lopez looks forward to celebrating her 10^th anniversary of living without seizures or medication

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Driving down a busy road, Roni-Kay Lopez knew she had to hurry and pull over.  Frightened, she couldn’t risk her life or the lives of others with what she knew was about to happen, so she drove through traffic to the side of the road. She then braced herself and suffered what she said felt like her millionth seizure.

After it passed and she started to regain her composure – she knew she couldn’t live like this anymore. The 29-year-old put her head on the steering wheel, exhausted. “They never took away my license, because I did feel them coming on and they were controllable.  But I wasn’t confident that would continue,” Lopez said.

Following that close call on the road, Lopez felt like enough was enough so she reached out to neurologist Selim Benbadis, MD, professor and director of the Comprehensive Epilepsy Program at USF Health and Tampa General Hospital. She wanted to find out if she was a candidate for surgery. She went through a thorough evaluation and after several tests, she found out her epilepsy was confined to the left temporal lobe of her brain. A few months later, neurosurgeon Fernando Vale, MD, also with USF Health, performed a left temporal resection on Lopez, removing a small part of the left temporal lobe.

Six weeks later, she begged to go back to work and she’s been going nonstop since.

USF Health neurosurgeon Selim Benbadis, MD, with patient Roni-Kay Lopez, who started the non-profit foundation Seize the Day to benefit people suffering from epilepsy.  Benbadis threw out the first pitch last year at a Rays game, where the foundation hosted a gathering.

Lopez looks forward to celebrating a milestone anniversary this year.  On April 24^th, she will be seizure and medication free for 10 years – the same day she went underwent surgery in 2008.

“If somebody would’ve told me as a kid, “You can do this, and you won’t have seizures.” I would’ve said, ‘yeah right, that’s not happening’. But, it’s happened and it’s been great,” Lopez beamed.

“This successful outcome with surgery is quite common with temporal lobe seizures, but unfortunately the delay before being evaluated for surgery is also very common,” Dr. Benbadis said of Lopez’s case.

Looking back, doctors told Lopez’s parents she would be lucky if she lived to be 16. Since infancy, she averaged almost 10 seizures a day. At times, her daily medications included up to eight tablets of Tegetrol (400 mg. each) and four Keppra (500 mg. each).

Lopez recalled a trip to the zoo with the Girl Scouts when she was 7 or 8 years old – she says she had 108 seizures that day. “It was really hot and humid and I guess the heat got to me,” she said. “My parents had to carry me around the zoo, because I refused to go home.”

Growing up with epilepsy was tough on her – many times she felt all alone.  “I got very tired. I would be doing something, have a seizure and lose all focus. I would try to get back to it, but many times I forgot what I was doing,” Lopez said. Besides seizures, another troublesome side effect included painful sores in her mouth.

Lopez, pictured here as a child, struggled with seizures for more than 20 years before her epilepsy evaluation and surgery by USF Health doctors.

In 2008, soon after the surgery, Lopez created Seize the Moment, a local non-profit foundation to help others who suffered from epilepsy. The foundation hosts many get-togethers, including outings at the Rays, Bucs and Lightning home games. “The Rays and the Lightning have been phenomenal in helping our foundation,” she said.

At a Rays game last year, Dr. Benbadis threw out the first pitch – a fitting tribute for someone who has helped so many.

In addition to hosting fun events for epilepsy patients and their families, the foundation also raises money with an annual bowl-a-thon and golf tournament. In February, Seize the Moment donated $10,000 to the Tampa General Foundation for funds raised in 2017.

To date, Seize the Moment has helped 12 patients and has provided aid to those with epilepsy, from ages 8 to 64.  “We want to put a smile on people’s faces — we are looking to make a difference,” Lopez said.  “In raising money, we aim to help with clinical research, patient care, medications and diagnostic testing.”

After going through so much the first 29 years of her life, Lopez needs only one word to describe how it feels to help her community, “Priceless!”

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-Story by Michelle Young and videos by Freddie Coleman, USF Health Communications and Marketing.
-Photos courtesy of Roni-Kay Lopez

A new device created at the University of South Florida – and including a cross-disciplinary team of experts from USF engineering, physical therapy and neurology – is showing early promise for helping correct the signature limp experienced by many stroke survivors.

Called the Gait Enhancing Mobile Shoe (GEMS), the shoe attachment is the result of multidisciplinary work and expertise in USF’s engineering, physical therapy, and neurology programs.

In addition to offering stroke patients good outcomes for improving their gait and balance, a preliminary study is showing the shoe also provides several advantages over a current stroke rehabilitation tool – the split-belt treadmill – including lower cost, greater convenience, and mobility.

“This is early in the process but we’re seeing the benefits we expected so it’s very promising,” said Kyle Reed, PhD, associate professor in the Department of Mechanical Engineering in the USF College of Engineering and principal investigator for the preliminary study on GEMS.

“We really want to help people who are limited in their walking ability to improve enough so they can return to the activities of their daily lives. The long-term hope is that this shoe attachment could be less expensive and safe enough that, once trained on how to use it, patients could take the GEMS home for therapy.”

Dr. Kim helps a patient try the GEMS shoe attachment.

Reed developed the GEMS shoe along with Seok Hun Kim, PT, PhD, associate professor in the School of Physical Therapy and Rehabilitation Sciences in the USF Health Morsani College of Medicine and co-principal investigator for the GEMS study. In 2010, Dr. Reed received funding from the National Institutes of Health to conduct a clinical trial of a small group of stroke survivors trying the GEMS; the study is not for severe stroke survivors, but mild to moderate stroke survivors.

The study also includes USF Health stroke expert David Z. Rose, MD, associate professor in the Department of Neurology in the USF Health Morsani College of Medicine, who said he sees the GEMS as a great potential option for stroke patients to improve their mobility.

“Many stroke patients are devastated that their ability to walk on their own can be so limited, even around their own homes,” Dr. Rose said. “Early data for the GEMS is very promising and the next phases of study will really help us see its true potential.”

Many stroke patients develop an asymmetric gait because of damage to their central nervous system, resulting in difficulty moving their affected leg – they can’t extend their foot backward enough, which prevents natural pushing off into the swing phase experienced in an unaffected walk.

Typical stroke rehabilitation to improve gait symmetry involves using a split-belt treadmill that offers two independent belts operating at different speeds to exaggerate the asymmetry of the patient’s gait.

But an odd yet natural thing happens when patients leave the treadmill – their brain returns to a fixed-floor state and they regress, with many finding it difficult to recreate the gait correction on solid ground, a regression that is called an after effect.

While generally successful for improving stroke patients’ gaits, the split-belt treadmill is expensive, requires a dedicated space to house and a qualified staff to monitor sessions and, because of after effect, can require more time for patients to master the correction, said Seok Hun Kim, PT, PhD, associate professor in the School of Physical Therapy and Rehabilitation Sciences in the USF Health Morsani College of Medicine.

“The GEMS allows movement across any safe surface, thus ‘rewiring’ the brain to learn the new compensation technique for everyday walking, not just for when they are on the treadmill,” Dr. Kim said.

“The GEMS is generally worn on the unaffected side, helping the patient use their affected side to compensate for the irregular footing.”

While early results of this preliminary study are showing strong support for a successful approach to improving the gait of stroke patients, more detailed study with more patients will be necessary. Dr. Kim said a full study, one that compares to the current approach with the split-belt treadmill, is critical before clinicians adjust their approach.

Dr. Kyle Reed demonstrates the GEMS shoe.

Story by Sarah Worth, photos and video by Sandra C. Roa, USF Communications

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.

Tampa, FLA (Jan. 17, 2017) — The University of South Florida has launched a neurosciences initiative to integrate the latest treatment and research for a wide range of neurological and psychiatric disorders.

With the state-of-the-art USF Health Byrd Alzheimer’s Institute as its centerpiece and driving force, the new neuroscience initiative is being spearheaded by Dr. Harry van Loveren, vice dean of clinical affairs for the USF Health Morsani College of Medicine and chair of the Department of Neurosurgery and Brain Repair. Dr. van Loveren has a proven track record of highly effective administrative and clinical leadership skills, having served as interim dean of the USF medical school and developed one of its most successful departments. He is widely respected regionally and nationally as a top neurosurgeon and researcher with expertise in skull base surgery as well as microneurosurgical techniques.

Harry van Loveren, MD

The multidisciplinary initiative will bring together medical, surgical and research specialists from USF Health’s departments of neurology, neurosurgery and psychiatry – all dedicated to advancing treatment and prevention of brain, spinal cord and other nervous system disorders.

“The primary goal of this larger neurosciences initiative is to foster and coalesce interdisciplinary research collaboration among USF Health scientists looking at the brain in unique ways to accelerate new discoveries for a broad range of neurological conditions – including Alzheimer’s and related dementias, Parkinson’s, ataxias, epilepsy, multiple sclerosis and stroke – leading to improved treatment and prevention for the patients we serve,” said Dr. Charles Lockwood, senior vice president for USF Health and dean of the Morsani College of Medicine.

“Dr. van Loveren has the expertise and commitment to build a preeminent, strong neurosciences model that will provide an even greater infrastructure for moving neurological discoveries from bench to bedside,” Dr. Lockwood said.

By consolidating disease-specific areas of care for a wide spectrum of neurological conditions, the initiative is expected to strengthen collaborative, complementary research across USF neuroscience disciplines and allow patients to better access the care they need.

“We will build upon the success of the Byrd Alzheimer’s Institute in becoming a vital treatment and research center for Floridians affected by Alzheimer’s disease and other memory disorders,” Dr. van Loveren said.

“The neurosciences initiative will unify and coordinate all resources at the university for neurological and neuropsychiatric disorders so we can expand our neurosciences footprint and serve even more patients and their families. We envision USF Health becoming a national destination for all things related to brain disorders and brain health.

The endeavor will also help position USF to take advantage of new federal funding opportunities such as the National Institutes of Health’s BRAIN Initiative, a pioneering project aimed at revolutionizing the understanding of the brain and accelerating the development and application of new technologies for brain disorders.

                                                                                                                   -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, the Biomedical Sciences Graduate and Postdoctoral Programs, 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

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

USF stroke experts staff the TGH Stroke Center, providing patients in the Tampa Bay region with 24/7 access to more specialized stroke care, including on-site neurosurgeons and medical teams trained in neurocritical care.

TAMPA, FL (Dec. 8, 2014) – Tampa General Hospital is the first hospital on the west coast of Florida to be awarded Comprehensive Stroke Certification, the highest national designation from the Healthcare Facilities Accreditation Program, a national certification organization recognized by the federal Centers for Medicare and Medicaid Services.

The new state-of-the-art Bayshore Pavillion at TGH has a dedicated Stroke Unit, a Trauma Resuscitation Unit and full capability CT and MRI scan in the Emergency Department; dedicated neuroangiography suites; and a Neurosciences Intensive Care Unit, which is one of the largest in the country.

This elite designation means the hospital meets the highest standards for safety and quality and can treat patients 24 hours a day with the most complex state-of-the-art stroke care.  Tampa General earned the national designation by meeting 21 evidence-based measures of complex stroke care. Continuous on-site neurologists, neurosurgeons, diagnostic radiologists and medical teams trained in neurocritical care are ready to treat emergencies immediately with highly trained and qualified nursing staff. Studies show that stroke patients have better outcomes when these standards are met.

“This provides an external validation for what we’ve known for a long time,” said Dr. W. Scott Burgin, director of Tampa General’s Comprehensive Stroke Center and professor of Neurology and chief of the Cerebrovascular Division at the USF Health Morsani College of Medicine.

“We are providing the highest level of care for our patients at Tampa General. Every stroke is different, and sometimes there are complications that require extremely specialized care that can only be found at comprehensive centers with a team on hand 24 hours a day who are trained to handle those complications.”

USF Health’s Dr. Scott Burgin directs the TGH Comprehensive Stroke Center.

Tampa General is the fourth hospital in the state to receive this national designation for its stroke program. It also is one of 34 Florida hospitals designated as a state Comprehensive Stroke Center, and one of 124 that the Joint Commission certifies as a Primary Stroke Center.

“This certification signifies that Tampa General Hospital has demonstrated and is clearly committed to advancing stroke treatment through innovation and best practice – placing them amongst world leaders,” said Michael Zarski, chief executive officer of HFAP. “Hospitals that have established stroke centers have demonstrated improved treatment, better patient outcomes, and reduced costs. In addition, Comprehensive Stroke centers are a resource to both the community and neighboring hospitals who are not equipped to offer the same level of care.”

Strokes occur when blood flow is disrupted in the brain. A clot can block a blood vessel, or bleeding can occur. If brain cells can’t receive blood and oxygen, they start to die, causing permanent damage. About 795,000 Americans have a stroke each year, making stroke the 4th leading cause of death in the U.S.

A stroke requires immediate emergency treatment and every minute counts. You can use the “FAST” acronym to recognize warning signs of a stroke: “Face drooping, Arm weakness, Speech difficulty, Time to call 911.”

-Tampa General Hospital-

Tampa General is a 1018-bed academic medical center on the west coast of Florida that serves as the region’s only center for Level l trauma, comprehensive burn care, and adult solid organ transplants. It is the primary teaching hospital for the USF Health Morsani College of Medicine. TGH is a comprehensive stroke center and a state-certified spinal cord and brain injury rehabilitation center. For more information, go to www.tgh.org.

Friedreich’s Ataxia Research Alliance and University of South Florida host Sept. 5^th event

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Tampa, FL (August 28, 2013) – Leading scientists and clinicians searching for a treatment for Friedreich’s ataxia (FA) and related disorders will gather for the fifth annual scientific symposium “Understanding a Cure,” 6 to 8 p.m., on Thursday, Sept. 5, at the USF Health Center for Advanced Medical Learning and Simulation, or CAMLS, 124 South Franklin Street, Tampa, FL  33602.

The symposium, free and open to the public, is hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the University of South Florida (USF) Ataxia Research Center.  Speakers will share the latest advances in research — from harnessing stem cell technology to investigating the effectiveness of a new medication.

The event routinely draws patients with Friedreich’s ataxia and their families 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, held on Saturday, Sept. 7.

For the second year in a row, the scientific symposium will be broadcast through the FARA Facebook page, with opportunities for visitors to join the conversation long distance.  To watch the presentations in real-time, visit https://www.facebook.com/CureFA/app_196506863720166 on Sept. 5 at 6 p.m. EST.  A Facebook account/membership is not needed to join.

Friedreich’s ataxia is a rare, debilitating neuromuscular disorder. Symptoms, emerges either between ages 5 and 15 or in adulthood and can progress to severe disability and include the following: loss of coordination and muscle weakness that leads to wheelchair use, energy deprivation and fatigue, vision impairment, hearing loss, slurred speech, aggressive scoliosis, diabetes, and life-shortening cardiac disease. There is not yet an approved treatment or a cure.

At last year’s symposium, Guy Miller, MD, PhD, CEO of Edison Pharmaceuticals Inc., announced that USF would lead a multisite study testing the safety and effectiveness of a potent antioxidant, the investigational drug known as EPI-743, for patients with Friedreich’s ataxia. Dr. Miller will return this year to report on the progress of that ongoing Edison-funded clinical trial, led by Theresa Zesiewicz, professor of neurology at USF Health.

Dr. Zesiewicz will join the discussion about the EPI-743 trial and update attendees on several other studies being conducted by the USF Ataxia Research Center.

Symposium speakers will include Mirella Dottori, PhD, principal investigator and senior research fellow at the Centre for Neuroscience Research, University of Melbourne, Australia, who will talk about //www.youtube.com/watch?v=TnLGtN8RVR4

Friedreich’s ataxia treatments with stem cells. Using skin cells from Friedreich’s ataxia patients converted into pluripotent stem cells, Dr. Dottori’s team recently induced these stem cells to generate the specific cell types that degenerate in Friedreich’s ataxia, including heart and nerve cells.

FARA President Ron Bartek and Jennifer Farmer, FARA executive director, will address progress nationwide in the research and management of Friedreich’s ataxia.

Dr. Zesiewicz will moderate a question-and-answer session on patients’ perspectives of living with ataxias, and Clifton Gooch, MD, chair of neurology at USF Health, will provide closing remarks.

USF is one of 10 sites included in FARA’s Collaborative Clinical Research Network, an international network of centers that share data and resources to advance treatments and clinical research for people with Friedreich’s ataxia.

For more information, please visit http://www.curefa.org/energyball/sep5.html 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 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

       – 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.

Video produced by USF Health Communications