The new office will allow the previously separate offices to combine resources and elevate their research efforts to improve health care for all Floridians

TAMPA, Fla (Nov. 30, 2020) — Tampa General Hospital and USF Health today announced the creation of a joint TGH-USF Health Office of Clinical Research to strengthen and expand current jointly conducted clinical trials, including translational studies that bridge laboratory discoveries and benefit patient care.

Both organizations are working to create Florida’s leading academic medical center dedicated to outstanding patient care, education and research. The restructuring will allow TGH and USF Health to combine resources and work together more seamlessly to initiate, operate and coordinate clinical trials looking at new ways to prevent, detect and treat disease.

“We already have established and invested in a strong foundation for clinical research,” said Tampa General President and CEO John Couris. “Combining the efforts of TGH and USF Health is the next step to elevate the world-class research we do to push forward to the edge of scientific discovery.”

“The joint office will allow for expansion of that portion of our clinical research portfolio occurring at TGH and to conduct that research with greater efficiency,” said Charles J. Lockwood, MD, MHCM, senior vice president of USF Health and dean of the USF Health Morsani College of Medicine.  “A robust research portfolio is a core component of all academic medical centers – and clinical trials are an essential part of what we do to advance the science leading to evidence-based health care. We expect this joint office to streamline the clinical trial process, thereby providing greater opportunities for both hospitalized patients and outpatients to participate in leading studies investigating new treatments.”

The joint TGH-USF Health Clinical Research Office will build upon the success over the last seven months of researchers and research staff at both institutions working to collaboratively launch about 35  COVID-19 clinical trials investigating a range of diagnostics, antiviral and anti-inflammatory medications, treatment protocols, vaccines and surveillance registries. Several, such as the joint studies testing Regeneron’s combination monoclonal antibody therapy in sick people or those exposed to the virus, are part of larger national clinical trials.

Including those COVID trials, TGH and USF Health now are working together on about 350 research studies. In July, TGH and the University of South Florida signed a new clinical affiliation to further solidify one of the largest academic medical centers in Florida and build upon their longstanding relationship and commitment to improving health care in Tampa Bay.

Clifton Gooch, MD, professor and chair of neurology at USF Health, was appointed co-vice president of Clinical Trials and Translational Research, and Rachel Karlnoski, PhD, was named executive director of the joint TGH-USF Health Office of Clinical Research.

This move to better align joint clinical research includes new leadership appointments:

• Clifton Gooch, MD, and Abraham Schwarzberg, MD, were named as co-vice presidents of Clinical Trials and Translational Research for the joint office. Dr. Gooch is professor and chair of the Department of Neurology at the USF Health Morsani College of Medicine and Tampa General Hospital Endowed Chair in Neurology. Schwarzberg is senior vice president of network development and chief of Oncology at TGH.

• Following a national search, Rachel Karlnoski, PhD, director of clinical research operations for USF Health, was selected to fill the new role of executive director of research. Karlnoski will report to Gooch and Schwarzberg for the oversight of all clinical studies involving both USF Health and Tampa General Hospital. She retains the position directing clinical research operations for USF Health, which she had held since 2018. For all USF Health trials except those based at TGH, Karlnoski continues reporting to Stephen Liggett, MD, vice dean for research at USF Health.

The administrative research changes will not affect USF Health’s participation in clinical studies with Moffitt Cancer Center, James A. Haley Veterans’ Hospital or other community and academic partners. Nor will the changes affect TGH’s partnership in clinical studies with Moffitt Cancer Center, TeamHealth, the Florida Orthopedic Institute, or other private practice physician partners.

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The age of human gene therapy has at last begun in earnest. This new era presents both tremendous opportunities and hope for our patients, but also brings unprecedented challenges, as accelerated discoveries and expensive new treatments are creating new clinical, political and ethical challenges.

I’ll share some USF Health research efforts to advance gene therapy for neurological diseases – but, first, some context.

Following the discovery of the structure of DNA in the 1950s, medical researchers predicted we would soon decipher the molecular mechanisms of hereditary diseases, enabling us to then genetically engineer treatments for those disorders shortly thereafter.  Although progress over the next several decades was far slower than anticipated, the launch of the Human Genome Project in 1990 made that future seem imminent.

However, in the late 1990s, the tragic death of a young patient volunteer (due to a systemic reaction to an adenovirus vector), along with a series of technical and clinical trial failures made effective gene therapy again seem like a distant dream.  Nevertheless, progress resumed in the mid-2000s and technological advances finally translated into effective clinical treatments over the ensuing decade.

In 2018, the FDA issued its first approval for a true in vivo gene therapy of a hereditary disorder (hereditary retinal dystrophy), and another for hereditary transthyretin amyloidosis, both of which are devastating diseases with no truly effective previous treatments.  These approvals were quickly followed by several more, including treatments and a possible cure for hereditary spinal muscular atrophy (SMA), a progressive and fatal paralyzing disease of infants and children.

Over the last year, a host of new therapies entered the pipeline for testing and FDA consideration. Many have shown robust and often dramatic Phase 2 and 3 evidence of efficacy.

Several USF Health Morsani College of Medicine (MCOM) researchers are on the front lines of these studies:

• Juan Sanchez-Ramos, MD, PhD, director of the USF Health Huntington’s Disease (HD) Center, received a $2 million NIH grant to develop and test a smart nanoparticle delivery system for placing gene therapies more precisely (and noninvasively) into areas of the brain affected by HD. He is also now launching an antisense oligonucleotide gene therapy study in HD patients. HD results from a triplicate repeat gene mutation, which mutates the huntingtin protein into a toxic form; this new therapy blocks production of the mutant protein, slowing or even arresting the disease.
• Theresa Zesiewicz, MD, director of the USF Health Ataxia Research Center, is working with a group of industry partners to develop gene therapy strategies for Friedreich’s ataxia (another triplicate repeat disorder).
• Tuan Vu, MD, director of the USF Health ALS Center, is exploring potential human studies of a gene therapy for one of the most common forms of hereditary amyotrophic lateral sclerosis (SOD1 ALS), in collaboration with the company that just secured FDA approval for its SMA gene therapy.
• Bob Hauser, MD, director of the USF Health Center for Parkinson Disease (PD) and Movement Disorders, is collaborating with a pharma company on genetic modulation of the production of alpha-synuclein (a pivotal protein in the pathogenesis of PD) in animal models, and to consider how this might be adapted as a therapy for human PD.

Many other outstanding MCOM translational and clinical researchers are now also entering this rapidly expanding arena.

With these advances and many more to come, this modern era of gene therapy continues to build excitement.  However, scientific progress has been so rapid that the world’s health systems are struggling to keep up with the integration and adoption of these new technologies. In particular, the cost of targeted gene therapies can impede their widespread use in clinical practice; pharmaceutical companies have priced many of these new therapies (some of which may actually be lifetime cures), at the multimillion dollar mark for each patient treated.

Beyond gene therapy, new genetic engineering technologies have profound implications for humankind. The world’s scientific, ethical and governmental bodies are now striving to quickly define where the boundaries of this technology should be, and grappling with when and how to discourage premature human experimentation in sensitive areas.

As controversy in other areas where science and politics intersect has demonstrated, surmounting these challenges together as a worldwide community may, in the end, be the ultimate hurdle to overcome as we enter the brave new world of gene therapy.

Clifton L. Gooch, MD
Professor and Chair, Department of Neurology
USF Health Morsani College of Medicine

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 Neuroscience Institute leaders accompany U.S. Rep. Gus Bilirakis on a tour of research laboratories in the Byrd Alzheimer’s Center, a centerpiece of the Institute.

Neurological diseases are growing at an unprecedented rate as Americans live longer and survive other chronic conditions like cardiovascular disease and cancer.

During U.S. Rep. Gus Bilirakis’ visit to the USF Health Neuroscience Institute on Aug. 24, the congressman was given an overview of the multidisciplinary research and clinical care that the University of South Florida conducts to help combat the human and economic burden of neurological diseases, including dementia, stroke, Parkinson’s disease and brain trauma.

The USF Health Neuroscience Institute, home of the Johnnie B. Byrd, Sr., Alzheimer’s Center, integrates three clinical departments — Neurology, Neurosurgery and Psychiatry – along with related basic and translational science departments. The Institute’s focus on consolidating disease-specific care for a wide spectrum of neurological diseases is intended to strengthen collaborative neuroscience research across USF and help accelerate promising discoveries from bench to bedside.

Laura Blair, PhD, assistant professor of molecular medicine, explains her team’s research on chaperone proteins that drive different states of the tau protein associated with Alzheimer’s and other neurodegenerative diseases. The USF Health researchers are identifying promising targets to help slow or prevent disease progression.

Bilirakis toured laboratories and the Johnnie B. Byrd, Sr., Alzheimer’s Center, the centerpiece of the Neuroscience Institute, with Stephen Liggett, MD, associate vice president for research at USF Health; Clifton Gooch, MD, chair of neurology at the USF Health Morsani College of Medicine (MCOM); and Glenn Currier, MD, chair of psychiatry and behavioral neurosciences at MCOM.  He also met with Robert Hauser, MD, director of the USF Parkinson’s Disease and Movement Disorders Center, which is designated a National Parkinson’s Foundation Center of Excellence.

These USF Health leaders spoke about the need for more federal resources directed toward helping advance early diagnosis and new treatments to delay, minimize and prevent nervous system disorders.

Robert Hauser, MD, (left) director of the nationally recognized USF Parkinson’s Disease and Movement Center, welcomes Bilirakis.

Among a few informational items they shared with the congressman:

• National Institute of Health grant funding does not cover the expense of high-tech equipment increasingly needed to do things like study single cells in the brain to understand the root cause of neurological diseases.
• 100 million Americans are afflicted with at least one neurological disease. A USF study led by Dr. Gooch and published last year in the Annals of Neurology found that the cost of diagnosed neurological disorders approaches a staggering $800 billion a year.
• More than half of MCOM’s $14.7 million in clinical trial revenue for FY 2018 was neuroscience related (neurology, psychiatry and neurosurgery).
• In addition to the Byrd Alzheimer’s Center and Parkinson’s Disease and Movement Disorders Center, other centers of excellence under the auspices of the USF Health Neuroscience Institute include stroke, epilepsy, multiple sclerosis (MS), Huntington’s disease, amyotrophic lateral sclerosis (ALS), ataxias with an emphasis on Friedreich’s ataxia, and aging and brain repair.
• USF Health Psychiatry and Behavioral Sciences continues to strengthen neurobiology research to discover new treatments for Alzheimer’s disease and abnormal brain development, as well as related mental disorders including autism. Current NIH-funded projects include studying ways to effectively deliver central nervous system drugs across the blood-brain barrier and testing the effectiveness of computer brain training games in protecting against cognitive decline.

USF Health’s Stephen Liggett, MD, (right) chats with Bilirakis in one of the laboratories at the Neuroscience Institute.

From left: Stephen Liggett, MD, USF Health associate vice president for research and Morsani College of Medicine vice dean for research; Congressman Gus Bilirakis, who represents Florida’s 12th District; Clifton Gooch, MD, chair of neurology; and Glenn Currier, MD, chair of psychiatry and behavioral neurosciences.

-Photos by Eric Younghans, USF Health Communications and Marketing

Clifton Gooch, MD, professor and chair of the USF Health Morsani College of Medicine’s Department of Neurology, has been selected president-elect of the Association of University Professors of Neurology (AUPN).  His two-year term begins May 1, 2018, and he will assume the role of president of the association for two years in May 2020, followed by a two-year position as immediate past president.

“I am greatly honored to have been elected by my fellow neurology chairs to help lead the AUPN over the next six years,” Dr. Gooch said.

The AUPN is the leadership organization for academic neurology, with membership consisting of all the chairs of neurology departments in accredited U.S. and Canadian medical schools. Neurology residency and clerkship program directors are also among the members. Founded in 1967 to foster the development of academic educational, research and patient care programs, the AUPN today also advises and influences policy affecting academic neurology at the national, state and local levels.

                                        Dr. Clifton Gooch

In 2016, Dr. Gooch spearheaded a national AUPN survey to assess the state of academic neurology in the United States during an era of unprecedented health care system reform.  The survey results were published July 2017 in Neurology, the journal of the American Academy of Neurology (AAN).  A second study led by Dr. Gooch detailing the enormous national cost of neurological diseases and a strategic plan for improving the neurological health of the nation was the second most highly accessed journal article published in the Annals of Neurology in 2017. It also was featured as a story in the Huffington Post.

Chair of the University of South Florida’s Department of Neurology since 2008, Dr. Gooch joined USF from Columbia University, where led the Neuropathy Research Center and the Neuromuscular Physiology Division.  During his tenure, the Neurology department has more than tripled in size and has greatly expanded its research portfolio, and now is conducting more than 100 clinical trials per year.  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.

In addition to his national leadership positions with AUPN, Dr. Gooch also serves on the Board of Directors for the American Neurological Association (ANA), has been a member of the FDA’s Nervous System Drug Approval Advisory Panel, and has provided expert testimony to Congress on improving the federal drug approval process.  A fellow of both the AAN and the ANA, he is highly sought as a speaker by many organizations across the country and internationally.

University of South Florida  study shows research investment critical to prevent destabilizing economic impact

TAMPA, FL (March 29, 2017) — More Americans are living longer and surviving chronic conditions like heart disease and cancer. Ironically, this triumph is also leading to a drastic rise in neurological disorders, which disproportionately attack the elderly.

Clifton Gooch, MD, professor and chair of the Department of Neurology at the University of South Florida Morsani College of Medicine in Tampa, is the lead author of study that details the enormous cost of neurological diseases to the nation.  The study is reported in the Annals of Neurology, the official journal of the American Neurological Association and the Child Neurology Society.

Clifton Gooch, MD

Working with USF College of Public Health colleagues Etienne Pracht, PhD, and Amy Borenstein, PhD, Dr. Gooch looked at the nine most prevalent and costly diagnosed neurological disorders and found the annual cost is staggering, totaling nearly $800 billion. By 2030, $600 billion will be spent treating stroke and dementia alone.  In addition, low back pain, traumatic brain injury, migraine headache, epilepsy, multiple sclerosis, spinal cord injury and Parkinson’s disease emerged as the most common disorders posing a serious financial burden.

“Given these extraordinary and rapidly growing costs, a concrete strategy is urgently needed to reduce the burden of neurological disease,” he said.

In the paper, Dr. Gooch calls on the federal government to provide more NIH funding to speed the development of treatments and cures for diseases such as dementia and stroke, including therapies to delay, minimize and prevent them. He also proposes the creation of a more effective national database to track treatment successes and failures.

“The very future of the neurological sciences and the patients we serve is now at stake, and the welfare of generations yet to come hangs upon the success of our efforts.”

Dr. Gooch writes that the years of productivity lost in the 100 million Americans living with neurological and musculoskeletal disorders is more than any other category of disease.

-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. The University of South Florida, established in 1956 and located in Tampa, is a high-impact, global research university dedicated to student success. USF is ranked in the Top 30 nationally for research expenditures among public universities, according to the National Science Foundation. For more information, visit www.health.usf.edu

Media contact:
Tina Meketa, University Communications and Marketing
tmeketa@usf.edu or (813)955-2593

Hosted by FARA and the USF Ataxia Research Center, the annual scientific symposium emphasizes a patient-centered approach to research

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

For the first time in its seven-year history, the Friedreich’s Ataxia Scientific Symposium brought together several pharmaceutical industry leaders to discuss preclinical and clinical studies.  The companies are all attacking Friedreich’s ataxia (FA) on different fronts with the same goal in mind:  to get the first treatment for the rare, but devastating, neuromuscular disease approved and on the market as soon as possible.

The Sept. 17 symposium, hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the USF Ataxia Research Center, drew an audience totaling more than 500, both live at the USF Marshall Center Ballroom and viewing the event in real-time through Ustream’s CureFA channel.

The symposium featured a discussion by leading representatives of pharmaceutical and biotechnology companies working with FARA and academia to conduct new research attacking Friedreich’s ataxia on several fronts.

Representatives and supporters of FARA, the research community, and patient and their families – many whom attended FARA’s Energy Ball gala on Saturday evening, Sept. 19 — were welcomed by USF President Judy Genshaft.

The translational center of excellence at USF is “one of the most active in the world, testing potential new drugs for Friedreich’s ataxia,” President Genshaft said. “We are unstoppable in the fight for a cure for FA.”

In the last seven years, the staff of the USF Ataxia Research Center, focused on identifying and developing effective treatments for inherited ataxia disorders, has expanded to eight, including two clinicians, a fellow, nurses and research coordinators.  Center Director Theresa Zesiewicz, MD, professor of neurology, gave an overview of the center’s eight clinical trials — six active and two finishing up. USF, one of 10 sites in the international FARA Collaborative Clinical Research Network, is recruiting patients for two of the three trials presented by pharmaceutical industry leaders at the symposium. The USF center is also working with Agilis, one of the biopharmaceutical companies at the symposium, to develop their gene therapy protocol, expected to be submitted in 2016 to the Food and Drug Administration (FDA).

USF President Judy Genshaft welcomed attendees to the USF Tampa campus for the seventh annual scientific symposium “Understanding Energy for A Cure,” hosted by the Friedreich’s Ataxia Research Alliance and the USF Ataxia Research Center.

Friedreich’s ataxia is triggered by a single genetic defect that limits production of frataxin, a protein vital to the function of the energy-producing factories, or mitochondria, of the cell. This leads to a variety of symptoms including neurodegeneration that can cause muscle weakness and loss of coordination and balance, energy deprivation and fatigue, vision impairment, slurred speech, aggressive scoliosis, diabetes and life-shortening cardiac disease. Most young people diagnosed with Friedreich’s ataxia require a cane, walker or wheelchair by their teens or early 20s. There are currently no approved treatments.

FARA President Ron Bartek and Executive Director Jennifer Farmer spoke about the progress in Friedreich’s ataxia research worldwide and the value of the organization’s 2,600-member patient registry in bringing together all its stakeholders.

FARA’s Executive Director Jennifer Farmer and President Ron Bartek.

Featured speaker Sanjay Bidichandani, MBBS, PhD, the chair of pediatric medical genetics at the University of Oklahoma College of Medicine and member of the FARA Board of Directors, was part of the team that first identified the Friedreich’s ataxia gene in 1996.  Fueled by resources and partnerships cultivated by FARA, Dr. Bidichandani said, the understanding of the disease process advanced relatively quickly since that genetic discovery and has yielded robust expansion of investigational drugs in the treatment pipeline.

A panel moderated by Dr. Bidichandani featured representatives from four biotechnology and pharmaceutical companies conducting Friedreich’s ataxia research in collaboration with FARA and academia – Jeffrey Sherman, MD, executive vice president for research and development and chief medical officer, Horizon Pharma; Jodi Cook, PhD, vice president of operations, Agilis Biotherapeutics; Colin Myer, MD, chief medical officer, Reata Pharmaceuticals; and Robert De Jager, MD, chief medical officer, Retrotope. Their newly activated studies cover a range of therapeutic targets, including finding ways to boost frataxin production, improving mitochondrial function, reducing mitochondrial damage and oxidative stress, and delivering gene therapy.

Featured speaker Dr. Sanjay Bidichandani, a member of FARA’s Board of Directors, was part of the group that discovered the gene for Friedreich’s ataxia in 1996. He gave an overview of the tremendous progress in less than 20 years leading to a robust treatment pipeline.

“FARA has really galvanized the patient community, academia, industry and even regulators to develop better insights into this disease and how we can all work together,” said Horizon Pharma’s Dr. Sherman. “At the end of the day, they’ve really brought to the forefront the importance of patient centricity and the voice of the patient in clinical and basic science research.”

Horizon Pharma has repurposed a drug already approved by the U.S. Food and Drug Administration for use in treating two other rare genetic disorders, chronic granulomatous disease and severe malignant osteopetrosis. The company recently launched a Phase 3 randomized, double-blind, placebo-controlled trial to test the safety and effectiveness of Actimmune® (interferon gamma-1b) in improving neurological function in 90 Friedreich’s patients at four U.S. sites. Previous research indicated that Actimmune®, which mimics a protein made by the body to help prevent infection, increases frataxin levels to reduce nerve cell depletion and muscle atrophy.

The symposium brought together representatives and supporters of FARA and the research community with patients in their families.

Agilis focuses on engineering and delivering therapeutic DNA to replace the damaged frataxin gene. Preclinical studies are employing a safe virus to optimally deliver the corrective gene to key targets, allowing safe and effective long-term expression of the frataxin protein.

Earlier this year Reata began a Phase 2 randomized, placebo-controlled, double-blind trial testing the safety and effectiveness of various dose levels of the oral medication RTA 408 in treating Friedreich’s ataxia. Known as the MOXle study, the trial is enrolling patients at sites worldwide, including USF. Preclinical studies have shown that RTA 408 directly activates antioxidative pathways to improve mitochondrial function.

This month USF enrolled the first of 18 patients in Retrotope’s 28-day randomized, double-blind controlled trial evaluating the safety of the investigational oral drug RT001 in ambulatory patients with Friedreich’s ataxia. USF and the University of California Los Angeles will be the only two sites for the Phase 1 study. The compound RT001 is a stabilized fatty acid shown to shut down the toxic free radical degradation of polyunsaturated fats, an essential component of cell membranes, and reduces further damage to the mitochondria.

Jade Perry, a member of the patient panel, was accompanied by her service dog Bo, a labradoodle.

The scientific panel was followed by a question-and-answer session on patients’ perspectives of living with Friedreich’s ataxia.  The panel was moderated by symposium host Clifton Gooch, MD, professor and chair of the Department of Neurology at the USF Health Morsani College of Medicine.

The four patient participants, all diagnosed in their teens or early 20s, emphasized a common theme – that they choose every day to carry on and live life to its fullest  despite the challenges of Friedreich’s ataxia.

Jade Perry, 25, was accompanied to the stage by the service dog she trained, a labradoodle named Bo.   Perry, who is finishing up a master’s degree in education from Coastal Carolina University, recently got her first full-time teaching job.

“I love riding my trike and try to keep my schedule packed so FA can’t slow me down,” she said.

Kendall Harvey, center, was diagnosed with later-stage Friedreich’s ataxia three years ago at age 25. “In spite of my diagnosis, my family and I are living life to the fullest,” she said.

Kendall Harvey, 28, diagnosed at age 25 with later-onset Friedreich’s ataxia, still walks unassisted.  She and her husband live in Austin, TX, with their 11-month old son Brooks.

Harvey, who participated in volleyball, track and other sports as a youth, said she noticed problems with agility and balance while taking dance lessons in preparation for her 2013 wedding.  She chalked it up to getting a little older or “being out of shape.”  But continuing symptoms finally led her to a neurologist who ran a battery of tests, culminating with a full genetic panel.

“The first time I heard of FA was the day I was diagnosed,” Harvey said. “FA has changed my perspective. Now, I live more in the moment than worrying about all the things in the future.

“On the days when I stumble a little more, my speech is slurred and I’m angry that my body is not behaving the way I’d like, my son is a fantastic reminder that I’m still capable of amazing things.  He’s very humbling.”

In his closing remarks Dr. Clifton Gooch, professor and chair of neurology at the USF Health Morsani College of Medicine, encouraged continued collaboration in the fight to find effective treatments and a cure for Friedreich’s ataxia.

USF’s Dr. Gooch closed the symposium by emphasizing the tremendous progress made in the research and development of lead drug candidates for Friedreich’s ataxia and encouraging all to continue to carry on the fight against the disease with laser focus.

“When pharma becomes engaged, that means the research is good enough to put smart money behind the disease to get a drug to market,” Dr. Gooch said. “There is more than just a glimmer of hope. We’re on the cusp of great possibilities… We look forward to the day when Friedreich’s ataxia will become a historical footnote like smallpox.”

For more information on the FARA patient registry, which provides notices about new clinical trials, go to http://www.curefa.org/patient-registry

Dr. Gooch, second from left, moderated the question and answer session with patients, l to r, Jade Perry, Erin O’Neil, Kendall Harvey, and Chris Nercesian.

FARA ambassador Kyle Bryant with Dr. Theresa Zesiewicz, director of the USF Ataxia Research Center.

Kyle Bryant with Sam Bridgman, who recently received a full scholarship to attend the graduate program at the USF Muma College of Business.

L to R: FARA President Ron Bartek; Dr. Clifton Gooch, chair of USF Health Neurology; Dr. Jodi Cook, vice president of operations, Agilis Biotherapeutics; Dr. Robert Molinari, founder and CEO of Retrotope; Dr. Jeffrey Sherman, CMO, Horizon Pharma; Dr. Theresa Zesiewicz, director, USF Ataxia Research Center; and Dr. Colin Myer, CMO, Reata Pharmaceutical.

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

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

They study, treat, and support every aspect of ALS, so it’s only fitting that the researchers, clinicians and staff from the USF Health Department of Neurology would team up to participate in the ALS Ice Bucket Challenge, a world-wide push to raise awareness and funds for ALS (amyotrophic lateral sclerosis), commonly called Lou Gehrig’s disease.

//www.youtube.com/watch?v=LTyU55GjB_Q

The ALS Ice Bucket Challenge began nearly three weeks ago when 29-year-old Pete Frates, who has ALS, posted a video challenging others to pour buckets of ice water over their heads within 24 hours. The video went viral, leading other participants – including celebrities, CEOs, and politicians – to shoot videos of themselves dumping ice buckets and then challenging friends to do the same within 24 hours or donate to the ALS Association (people tend to do both). The effort has reached millions across social media and raised more than $31.5 million for ALS, as of Aug 20.

USF Health is home to the USF Health ALS Center, which provides multidisciplinary comprehensive care to ALS patients and conducts research seeking new treatments and a cure.

As for this USF Health Neurology Ice Bucket Challenges, nearly 30 people, each with a bucket filled with ice water in front of them, gathered on the steps in front of the Carol & Frank Morsani Center for Advanced Healthcare. After a brief introduction by Emily Wingate (daughter of an ALS patient at the USF ALC Center), Dr. Clifton Gooch (chair of Neurology in the Morsani College of Medicine), Dr. Tuan Vu (director of the USF Health ALS Center), and Dr. Lara Katzin (co-director of the USF Health ALS Center), and a count to three, they all lifted buckets and poured the freezing wet contents over their heads to a roar and applause from onlookers and some cheers of “Go Bulls!”

As is part of the Ice Bucket campaign – to challenge others to participate – the group challenged the student body of the USF Health Morsani College of Medicine.

Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease) is a deadly disease that progressively paralyzes its victims, attacking nerve cells and pathways in the brain and spinal cord. Patients are robbed of the ability to walk, eat, speak, and eventually, breathe, and most live for just two to five years after diagnosis.

Witnessing the fun was Gina Rathbun, director of physician and community relations for the USF Physicians Group, who is a patient in the ALS Center.

“We want the funds raised from this kind of event to go to research, which has always been underfunded,” Rathbun said. “Those funds allow for more clinical trials, which are critical to finding answers.”

Participating in the USF Neurology ALS Ice Bucket Challenge included: Dr. Clifton Gooch, Dr. Tuan Vu, Dr. Lara Katzin, Lise Casady, Dr. Frank Delgado, Dr. Rebecca Edgeworth, Brittany Golden (ALS Clinic Co-Coordinator), Juliana Gordon, Hiram Green, Ieisha Hall, Kristin Huynh, Linda Jones, Nichole Jones, Stephanie Kelley, Heather Klein, Jennifer Kuenning, Theresa McClain, (ALS Clinic Nurse Practitioner), Eileen Massey, Vicky Mastorides, Ashley Meeke, Jessica Moret, Vanessa Rivera, Joseph Staffetti, Carshena Tisdale, Natalie Tucker (ALS Clinic Coordinator), Dexter Werner,  Emily Wingate, Kristin Winkler, Dr. Eric Vernier, and Kayla Zayas.

If you would like to support research at the USF ALS Clinic, click here to donate now.

Local media attended the event and got Dr. Gooch’s reaction to the cause.

They all punctuated the event with a Go Bulls!

Co-organizer of the Neurology ALS Ice Bucket Challenge Brittany Golden

Video by Jesse McLane

Photos by Rebekah Wright, USF Health Morsani College of Medicine

Story by Sarah Worth, USF Health Office of Communications

Increased funding helps provide much-needed coordinated care to ALS patients and families across Florida. Access to care truly made a difference to Billie Jean Fogle. 

There’s no cure for the debilitating terminal disease known as Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease), but access to comprehensive ALS care extends and improves patient’s lives, and also enhances the lives of their families. To help residents throughout Florida gain access to this care, the Florida Legislature passed the Bitner/Plante Amyotrophic Lateral Sclerosis Initiative of Florida during the 2013 session to provide $1 million to greatly expand services at those Florida clinics offering the highest level of comprehensive ALS Care.

ALS patient Billie Jean Fogle (left), was relieved to have access to personalized, comprehensive care, said her daughter Teresa Zeller (right). Photo courtesy of Teresa Zeller.

“This is a direct benefit to the patients and their families,” said Clifton Gooch, MD, professor and chair of the USF Health Department of Neurology and an ALS specialist. “Research has proven that a multidisciplinary approach to care prolongs the lives of ALS patients and improves the quality of their lives. However, the barrier for most ALS patient is access to that care, as our current medical system does not provide coverage for many of the key components of this treatment.”

The funding allowed the USF Health ALS Center, which opened October 2011, to double the number of patient appointments and to significantly shorten the patient wait list. The funding also allowed for similar expansions at the other comprehensive ALS centers sites in Florida, including the University of Miami, University of Florida Shands in Jacksonville, and the Mayo Clinic Jacksonville.

To help disperse care even further across Florida, the initiative is also pioneering a telemedicine component, using technology to provide visual interaction for patients with the many providers necessary to treat their disease.

Dr. Clifton Gooch at the opening of the USF Health ALS Center in 2011.

“The telemedicine component means that even ALS patients living in rural areas who can’t make it to one of the centers for their many appointments can now be monitored and treated between face-to-face clinic visits,” Dr. Gooch said.

The initiative will also facilitate more research toward improved treatment by increasing the number of patients enrolling in clinical trials.

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Teaming up makes the difference to ALS patients
ALS is a deadly disease that progressively paralyzes its victims, attacking nerve cells and pathways in the brain and spinal cord. Patients are robbed of the ability to walk, eat, speak and, eventually, breathe. Most live for just two to five years after diagnosis.

ALS patients require care from many specialists, including neurologists, speech therapists, physical therapists, respiratory therapists, nutritionists, and psychologists, among others. Coordinating the care within a single multidisciplinary team visit ensures patients will receive all the care that they need in a coordinated fashion. It also is much easier on debilitated ALS patients and their families, sparing them from making dozens of trips to separate appointments to see many specialists over time.

“A comprehensive multidisciplinary center provides under one roof all of the services ALS patients require, and that makes the biggest difference in the life of an ALS patient,” said Tuan Vu, MD, professor of neurology and director of the USF Health ALS Center.

“Funding allows us to bridge the access gap and provide much needed care for ALS patients,” Tuan Vu, director of the USF Health ALS Center.

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How big of a difference?
There was about three years between a diagnosis of ALS and death for Billie Jean Fogle.

But that short time was made much more comfortable – and maybe even prolonged – because of the ALS Center at USF Health, said Fogle’s daughter Teresa Zeller.

“Finding the USF Health ALS Center was wonderful,” Zeller said. “Traveling is so hard, nearly impossible really.”

Zeller said her mother connected with USF in April 2011 and was greatly relieved to have such personalized care.

“The biggest blessing for Mom was that the team was all there at one appointment, all of them in one room. They would go in and out of the room to talk to us, we didn’t move from specialist to specialist. It was a long morning for Mom but so much better than another clinic we visited. It was all in one place, but we were the ones who had to move within the building.”

The team approach is what really made the difference, Zeller said.

“They all worked together, instead of piecing together her care,” she said. “And the support staff really helped us follow up on the orders that came out of her appointments. We didn’t have a moment to make five phone calls to look into her respiratory therapy. They told us exactly who to contact.”

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Building an initiative to improve access to ALS care
The impetus for seeking funding came when Wendy Bitner toured the USF Health ALS Center through the ALS Association’s Hope and Help Tours. Her husband, Florida State Rep. David Bitner, had recently died of ALS in September 2011.

When she asked how she could help, Dr. Gooch described the difficulty the Center has in securing funding every year and that the current annual funding allowed for a limited number of appointments for ALS patients. Bitner, together with Dr. Gooch and members of the ALS Association, framed a proposal for $1 million that would not only expand the ability of USF Health’s ALS Center to take additional patients but would also fund expansions at three additional centers across the state to help increase access to treatment and support for ALS patients throughout Florida.

David Bitner was highly regarded and his colleagues in Tallahassee did not hesitate to help shepherd the proposal through. The proposal was approved by the Florida Legislature in Spring 2013.

ALSA is administering the project and collecting data on the number of patients being seen at the four clinics.

Administration of the Initiative is unique: there is no single person charged with directing the Initiative, but a group of ALS experts from each of the centers instead guides the program.

“We are very democratic, and have created this round table approach, so that everyone has equal input,” Dr. Gooch said. “We all want to work together in an equitable way and avoid problems that come from decisions being based at one facility over another.”

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Looking ahead
The hope, Dr. Gooch said, is that state elected officials will see the value in this and help grow the funding.

“We need to keep all of these centers open and running and spread these benefits all over the state,” Dr. Gooch said. “The academic comprehensive ALS centers participating in this initiative are unique because they are able to provide the highest level of expertise and the most comprehensive levels of care. ALS operations of this kind take years to develop and rely heavily on the resources and infrastructure of the academic medical center, which is why there are a limited number of centers.”

But continued funding will mean continued access to care and an increase in research opportunities, he added.

“The patient-care side of these ALS centers is only part of the operation,” Dr. Gooch said. “There is also a great deal of translational research, offering patients everywhere hope for a cure.”

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How you can help
One way you can help is to join the local Walk to Defeat ALS, set for 9:30 a.m. Saturday, March 8, 2014. Click here for more information.

Dr. Clifton Gooch addressing supporters at the annual ALS Walk.