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The Friedreich’s Ataxia Scientific Symposium: Understanding a Cure recently marked its 9th year of bringing dedicated scientists, clinicians, and industry partners with patients and their families to USF – all whom share a commitment to finding effective treatments and a cure for Friedreich’s ataxia and related disorders.  The Tampa Bay region is considered an epicenter for raising funds to support  research for Friedreich’s ataxia, or FA.  This year’s 10th Friedreich’s Ataxia Research Alliance (FARA) Energy Ball, held Saturday, Sept. 29,  following the symposium and the association’s international FA biomarkers meeting, raised a total of $2.5 million to help bridge the gap between discoveries and treatment.

Hosted by FARA and the USF Ataxia Research Center, the scientific symposium drew an audience of 700 — both live at the USF Health Center for Advanced Medical Learning and Simulation (CAMLS) and viewing the event in real-time through YouTube.

From left: Paul Avery, Dr. Theresa Zesiewicz, Dr. Charles J. Lockwood, Dr. Clifton Gooch, Jennifer Farmer, and Ron Bartek

FA is a rare inherited neuromuscular disease, typically diagnosed in children and adolescents, that progressively robs patients of their ability to walk, speak and function and can ultimately lead to early death. Currently, no drugs are approved for the treatment of FA.

Symposium participants discussed the latest FA translational research progress, as well as heard individual patient perspectives and from scientific and industry partners working with academia to move promising therapies into the clinical trial pipeline.  Researchers are attacking the devastating disease using multiple approaches, including working to improve mitochondrial function and inhibit inflammation, to replace the protein (frataxin) deficient in individuals with FA, and to combine gene correction with hematopoietic stem cell therapy. They also look for targeted biomarkers to help clinicians better assess disease progression and the response of individual patients to investigational FA drugs.

USF Health is one of 11 sites in Friedreich’s Ataxia Collaborative Clinical Research Network, an international network of clinical research centers working together to advance treatments and clinical care for those living with FA.  The USF Ataxia Research Center participates in the FA patient registry, the only worldwide registry containing demographic and clinical information on more than 2,000 patients with Friedreich’s ataxia.

Here’s a sampling of what some symposium speakers had to say:

Charles J. Lockwood, MD, senior vice president of USF Health and dean, USF Health Morsani College of Medicine

“Since 2008, this symposium continues to be a major annual event broadcast around the world… We will never stop looking for answers. We will never stop looking for a cure.”

Dr. Zesiewicz, center, with her USF Ataxia Research Center team

Theresa Zesiewicz, MD, professor of neurology and director, USF Ataxia Research Center

“At USF we have several active trials for FA, and we are close to finding a treatment to arrest disease progression and help alleviate some of the symptoms of FA. We are working towards offering even more clinical research in the future.”

From left, the Patient Panel, moderated by FARA Communications Director Felicia DeRosa, included: Laurel Avery of Florida, Sam Hill of Maryland, Annie Hamilton of New York, and Allison Dana of South Carolina.

Patient Panel:  Living with FA and Research Participation

Laurel Avery, 23, Tampa, FL, currently working on her master’s degree in entrepreneurship

“When I first started college, people automatically assumed that I need help with everything.  That’s a misconception, because the FA community is very adaptable and everyone works very hard to be resilient and strong.”

Sam Hill, 14, Annapolis, MD

“I’m proud that I’m able to keep up at school, even though I get tired easily.  I like math and science.”

Advocacy Organization Panel

Sanjay Bidichandani, PhD, FARA scientific director and head of genetics at the University of Oklahoma College of Medicine Department of Pediatrics

“From the beginning of when the FA gene was found until today, all the discoveries are now nodes or targets for therapies being developed.”

Advocacy Organization Panel participants included, from left: Sanjay Bidichandani, PhD, FARA scientific director; FARA President Ron Bartek; and Pat Ritschel, FARA board director

Ron Bartek, FARA president and co-founder

“We realized from the get-go that to develop a treatment and cure for this disease we’d need to build relationships with patients and their families, with academic investigators, with government partners at the NIH, FDA and Congress, and with industry partners.  With our encouragement, all of those stakeholders are now members of the family… Collaboration is FARA’s middle name.”

Pat Ritschel, FARA Board director

“By spending our effort to remove roadblocks, we attract more companies willing to work in this space of FA research… because the way forward is clear and they can see a path to the clinic and (drug) approval.”

The Scientific Panel included, sitting from left: Carole Ben-Maiman, MD, CEO of Chondrial Therapeutics; Stephanie Cherqui, PhD, associate professor at UC San Diego; Hao Wang, PhD, senior scientific director at Clinical Neuroscience-CNS TAU, Takeda Pharmaceuticals; and Kara Eichelkraut, senior manager of patient advocacy for Reata Pharmaceuticals.

Scientific Panel – FA Treatment Approaches

Jennifer Farmer, FARA executive director

“We have lots of different treatment approaches in various stages of development.  This is very important, because we know that there is no disease fully treated with just one drug or one therapy.”

Hao Wang, PhD, senior scientific director of Clinical Neuroscience-CNS TAU, Takeda Pharmaceuticals

“The patients have educated us about their diagnosis and journey, and what kind of treatments they need. They have a great sense of humor despite the difficulties they are experiencing… The feedback we received from them has helped with study design.”

(Takeda’s investigational drug candidate TAK-831 is in a phase 2 clinical study to evaluate the enzyme inhibitor’s effectiveness on upper extremity motor function and manual dexterity.)

Kara Eichelkraut, senior manager of patient advocacy, Reata Pharmaceuticals

“Anything and everything anyone in this room does to contribute to FA research makes a difference.  If it’s participation in a clinical trial that’s great, but if you cannot do that there are other things you can do, like providing a cheek swab or giving a vial of blood.”

(Reata received orphan drug designation for omaveloxolone for investigational treatment of FA by the FDA and EMA. FARA is enrolling 100 patients in a phase 2, part 2 clinical trial.)

Closing Comments

Clifton Gooch, MD, chair of neurology, USF Health Morsani College of Medicine, thanked participants for their courage and inspiration

“Twenty years after the founding of FARA we have over a dozen clinical trials focused on getting at the very heart of this disease in the genes themselves, using techniques of genetic engineering and modulation, therapies to support the mitochondria, and others… There is great reason for hope.

“Once we find the genetic therapy that treats this disease we will have the tools to prevent it in those who have the aberrant gene, to fix the gene so that they never develop the disease… That’s the world we’re working towards. We want to make FA a footnote in the medical literature, like smallpox.”

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-Videos by Torie M. Doll, and photos by Eric Younghans, USF Health Communications and Marketing

International experts from academia and industry will gather to discuss advances in Friedreich’s ataxia from the laboratory to the clinic.

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Tampa, FL (Sept. 12, 2016) — The University of South Florida (USF) will bring together leading researchers and patients searching for a treatment for Friedreich’s ataxia (FA) and related disorders at the eighth annual scientific symposium “Understanding Energy for A Cure.” The symposium will be held 5 to 8:30 p.m., Thursday, Sept. 15, at the USF Sam and Martha Gibbons Alumni Center, 11810 USF Alumni Drive, 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.

The symposium will include four scientific panel discussions moderated by FARA spokesperson Kyle Bryant. The topics will be:

• Basic and Discovery Science
• Drug Development and Advancing Treatments
• FA Biomarkers
• Clinical Trials and Translating Treatments to Improved Care

Before the scientific panel discussions, Dr. Theresa Zesiewicz, professor of neurology and director of the USF Ataxia Research Center, will update attendees on the Friedreich’s ataxia initiatives at the center. USF is one of 10 sites in the international FARA Collaborative Clinical Research Network.

Patients will share their experiences about living with FA in a discussion moderated by Jen Farmer, executive director of FARA.

Each year, the Friedreich’s Ataxia Scientific Symposium draws patients and their families 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. 17.

For the first time this year, on Friday Sept. 16, USF will also host FARA’s 4^th Biomarkers Meeting, which attracts international scientists to focus on measurable metabolic, neurological and cardiac indicators of Friedreich’s ataxia as well as endpoints for the disease.

Thursday’s “Understanding Energy For A Cure” symposium will be broadcast through the 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. 15 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.

For more information or to RSVP, please visit www.curefa.org/events/event/337-understanding-energy-for-a-cure-symposium, 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, the Biomedical Sciences Graduate and Postdoctoral Programs, and the USF Physicians Group. The University of South Florida is a Top 50 research university in total research expenditures among both public and private institutions nationwide, according to the National Science Foundation. For more information, visit www.health.usf.edu

                                                – 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

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