Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease affecting the nerve cells in the brain and spinal cord. The disease causes motor neurons to slowly degenerate, which causes the control of muscle movement and the brain’s ability to initiate that movement to be lost. This causes ALS patients to eventually lose the ability to speak, swallow, move, and even breathe.

The average life expectancy of individuals suffering from ALS is two to five years. However, consistent treatment and support from at an ALS clinic – with a multidisciplinary team that can provide the newest treatments, therapies and access to clinical trials – are proven to help slow the disease’s progression.

“ALS has different variants; some patients will have longer survival rates than others,” said Tuan Vu, MD, professor in the Department of Neurology in the USF Health Morsani College of Medicine and director of the USF Health ALS Clinic. “However, through supportive care, patients do live longer and there are studies showing that ALS clinics do prolong a patient’s life.”

The team at the USF Health ALS Clinic includes social workers, dietitians, and physical, speech, and respiratory therapists, as well as neurologists, nurse practitioners and primary care physicians. By bringing such a range of disciplines and experts together, the clinic can provide treatment all in one location at individual appointments, so patients do not have to make multiple appointments to see each provider. After each appointment, the team meets to discuss the patient’s current situation and coordinates next steps in care.

“Our meetings result in a much more seamless level of care that we would not be able to achieve otherwise,” Dr. Vu said. “As a team, the goal is to improve the quality of life of the patient and lessen the burden of the disease.”

Tina Orr is a patient at the USF Health ALS Clinic who has lived with ALS for three years.

Tina Orr visits the USF Health ALS Clinic.

“Here, they do not look at you as a number; they look at you as who you really are,” she said. “They will help you in any way possible. To have my voice, to be able to eat what I want, and be able to spend time with my family…They have made that possible for me.”

And Gina Rathbun was diagnosed with ALS in 2009 and has lived with the disease for more than 14 years.

David Rathbun and Gina Rathbun.

“All of these wonderful providers have helped us so much in adapting our life to live with this disease,” said David Rathbun, Gina’s husband. “Every time we go, we learn about what works and what doesn’t work. It’s been a really wonderful experience and incredibly helpful. Gina has been an inspiration to me and everyone around her.”

Currently, there is no cure for ALS. However, there are multiple efforts to produce effective treatment plans for patients. Recently, the U.S. Food and Drug Administration (FDA) approved Rilutek®, the first drug that has reliably extended the life expectancy of people with ALS. Despite this, the drug will not strengthen or restore patients’ strength. It is one of many new medications on the horizon that may help ease the burden for those facing ALS.

”Our hope is that when patients think they may have the condition, they reach out directly to our clinic,” Dr. Vu said. “We do make a concerted effort to reach back out to these patients as quickly as we can. The sooner we can implement treatment and involve patients in clinical trials that help slow down the disease the better they will be.”

For more information about the USF Health ALS Clinic and scheduling an appointment, call the Neurology scheduling line at 813-396-9478.

For more information on making a donation to the USF ALS Center please contact Jennifer Kuenning at 813-396-2523

Story and video by Ryan Rossy, USF Health Communications and Marketing

A new University of South Florida preclinical study finds that the regenerative cell therapy boosts motor nerve cell survival by repairing the blood-spinal cord barrier

TAMPA, Fla. (April 2, 2019) — Transplantation of human bone marrow-derived endothelial progenitor cells (EPCs) into mice mimicking symptoms of amyotrophic lateral sclerosis (ALS) helped more motor neurons survive and slowed disease progression by repairing damage to the blood-spinal cord barrier (BSCB), University of South Florida researchers report.

The study was published March 27 in Scientific Reports, one of the Nature journals. The findings contribute to a growing body of work exploring cell therapy approaches to barrier repair in ALS and other neurodegenerative diseases.

Human bone marrow-derived endothelial progenitor cells in vitro

The progressive degeneration of nerve cells that control muscle movement (motor neurons) eventually leads to total paralysis and death from ALS. Each day, an average of 15 Americans are diagnosed with the disease, according to the ALS Association.

Damage to the barrier between the blood circulatory system and the central nervous system has been recognized as a key factor in the development of ALS. A breach in this protective wall opens the brain and spinal cord to immune/inflammatory cells and other potentially harmful substances circulating in peripheral blood. The cascade of biochemical events leading to ALS includes alterations of endothelial cells lining the inner surface of tiny blood vessels near damaged spinal cord motor neurons.

This latest study by lead author Svitlana Garbuzova-Davis, PhD, and colleagues at the USF Health Morsani College of Medicine’s Center of Excellence for Aging & Brain Repair, builds upon a previous study showing that human bone marrow-derived stem cells improved motor functions and nervous system conditions in symptomatic ALS mice by advancing barrier repair. However, in that earlier USF study the beneficial effect was delayed until several weeks after cell transplant and some severely damaged capillaries were detected even after a high-dose treatment. So in this study, the researchers tested whether human EPCs – cells harvested from bone marrow but more genetically similar to vascular endothelial cells than undifferentiated stem cells – would provide even better BSCB restoration.

Svitlana Garbuzova-Davis, PhD

ALS mice were intravenously administered a dose of human bone-marrow derived EPCs.  Four weeks after transplant, the results of the active cell treatment was compared against findings from two other groups of mice:  ALS mice receiving a media (saline) treatment and untreated healthy mice.

The symptomatic ALS mice receiving EPC treatments demonstrated significantly improved motor function, increased motor neuron survival and slower disease progression than their symptomatic counterparts injected with media. The researchers suggest that these benefits leading to BSCB repair may have been promoted by widespread attachment of EPCs to capillaries in the spinal cord. To support this proposal, they point to evidence of substantially restored capillaries, less capillary leakage, and re-establishment of structural support cells (perivascular astrocytes) that play a role in helping form a protective barrier in the spinal cord and brain.

Further research is needed to clearly define the mechanisms of EPC barrier repair.  But, the study authors conclude: “From a translational viewpoint, the initiation of cell treatment at the symptomatic disease stage offered robust restoration of BSCB integrity and shows promise as a future clinical therapy for ALS.”

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

Article citation:
Svitlana Garbuzova-Davis, Crupa Kurien, Edward Haller, David J. Eve, Stephanie Navarro, George Steiner, Ajay Mahendrasah, Surafuale Hailu, Mohammed Khatib, Kayla J. Boccio, Cesario V. Borlongan, Harry R. Van Loveren, Stanley H. Appel and Paul R. Sanberg. Human Bone Marrow Endothelial Progenitor Cell Transplantation into Symptomatic ALS Mice Delays Disease Progression and Increases Motor Neuron Survival by Repairing Blood-Spinal Cord Barrier, Scientific Reports, March 27, 2019. https://doi.org/10.1038/s41598-019-41747-4.

ALS mice improved with stem cell therapy; first step for science in finding better treatment  

TAMPA, Fla. (May 12, 2017) – Researchers at the University of South Florida show in a new study that bone marrow stem cell transplants helped improve motor functions and nervous system conditions in mice with the disease amyotrophic lateral sclerosis (ALS) by repairing damage to the  blood-spinal cord barrier.

In a study recently published in the journal Scientific Reports, researchers in USF’s Center of Excellence for Aging and Brain Repair say the results of their experiment are an early step in pursuing stem cells for potential repair of the blood-spinal cord barrier, which has been identified as key in the development of ALS. USF Health Professor Svitlana Garbuzova-Davis, PhD, led the project.

Previous studies in development of various therapeutic approaches for ALS typically used pre-symptomatic mice. This is the first study advancing barrier repair that treats symptomatic mice, which more closely mirrors conditions for human patients, Dr. Garbuzova-Davis said.

Svitlana Garbuzova-Davis, PhD, led the study.

Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the process of blood-spinal cord barrier repair.

The stem cell treatment delayed the progression of the disease and led to improved motor function in the mice, as well as increased motor neuron cell survival, the study reported.

ALS is a progressive neurodegenerative disease that affects neuronal cells in the brain and the spinal cord, which send signals to control muscles throughout the body. The progressive degeneration of motor neuron cells leads to death from ALS. More than 6,000 Americans each year are diagnosed with the disease.

Because stem cells have the ability to develop into many different cell types in the body, researchers at USF’s Center of Excellence for Aging and Brain Repair, Department of Neurosurgery and Brain Repair have focused on using stem cells to restore function lost through neurodegenerative disorders or injuries.

Damage to the barrier between the blood circulatory system and the central nervous system has been recently recognized as a factor in ALS development, leading researchers to work on targeting the barrier for repair as a potential strategy for ALS therapy.

In this study, the ALS mice were given intravenous treatments of one of three different doses of the bone marrow stem cells. Four weeks after treatment, the scientists determined improved motor function and enhanced motor neuron survival. The mice receiving the higher doses of stem cells fared better in the study, the researcher noted.

The transplanted stem cells had differentiated into endothelial cells – which form the inner lining of a blood vessel, providing a barrier between blood and spinal cord tissue – and attached to capillaries in the spinal cord. Furthermore, the researchers observed reductions in activated glial cells, which contribute to inflammatory processes in ALS.

USF Health Morsani College of Medicine researchers Crupa Kurien, Avery Thomson, Dimitri Falco, Sohaib Ahmad, Joseph Staffetti, George Steiner, Sophia Abraham, Greeshma James, Ajay Mahendrasah, Paul R. Sanberg and Cesario V. Borlongan joined in the project. The study was funded by the National Institutes of Health, National Institute of Neurological Disorders and Stroke.

Read the full study here.

                                                                                                                                                     -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

News release by Vickie Chachere, USF Research and Innovation

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

//www.youtube.com/watch?v=qwe7-t-yleM

Jaymin Kathiriya, MS, an aspiring young researcher who investigates how hypoxia exacerbates pulmonary fibrosis, was among the select group of 11 students invited to give oral presentations at the 2016 USF Health Research Day.

Kathiriya, a fourth-year PhD candidate in the Department of Pathology and Cell Biology, was enthusiastic about the chance to present his research before student peers and faculty judges Feb. 19 in the Oval Theatre at the USF Marshall Student Center. Even more than that, he appreciated the opportunity to mingle with fellow aspiring young researchers from across all USF Health colleges and disciplines, as well as guest researchers from USF programs studying the science of health.

“I’m excited about being able to get critical input and different ideas from so many different people,” he said.

The energy of aspiring young researchers from across all USF Health colleges and disciplines generated a buzz in the Marshall Student Center Ballroom during the 2016 USF Health Research Day.

A training ground for aspiring researchers, physicians

Charles J. Lockwood, MD, senior vice president for USF Health and dean of the Morsani College of Medicine, served as a judge for this year’s Research Day. He said he was impressed by the depth and breadth of the research activities encompassed in the nearly 330 poster presentations.

“I have dedicated a significant portion of my career to my own research and to training physician-scientists – so I may be biased,” Dr. Lockwood said. “But, I strongly believe that, beyond its intrinsic importance in promoting health, research makes one a better provider by disciplining the mind to collect all the requisite data and then carefully and correctly interpret it to make the correct diagnosis and choose the optimal therapy.”

Dr. Charles Lockwood judges the work of Kristen Marcet, second-year medical student.

Phillip Marty, PhD, associate vice president for USF Health Research, agreed.

“Our faculty are engaged in important research, which translates directly to our students, graduate students, residents and trainees who are presenting here today,” Dr. Marty said. “It is a great training ground for the rest of their careers.”

This year’s slate of presenters included more students and trainees and slightly fewer faculty, Dr. Marty said, perhaps a reflection of the event’s return to its roots of showcasing science learners.

Paul R. Sanberg, PhD, DSc, Senior Vice President for Research, Innovation & Economic Development, said USF is committed to providing its students with research and commercialization opportunities to support their growth and development as scientists, leaders and, ultimately, mentors to others.

“The accomplishments of your faculty, students, trainees and staff at celebrated at Research Day are more examples of USF Health’s continued impact in research and innovation,” Dr. Sanberg said.

Nearly 330 students and faculty presented their research projects in a judged competition.

Student contributions to scientific body of knowledge

In Dr. Dave Vrushank’s laboratory, Kathiriya works with cell and animal models to help investigate the contribution of low oxygen levels, or hypoxia, to pulmonary fibrosis of unknown cause, known as idiopathic pulmonary fibrosis (IPF). IPF is an incurable disease in which tissue deep in the lung becomes scarred over time. The prevalence of this incurable disease, which kills up to 40,000 Americans a year, has increased as the aged population has grown.

“We are trying to determine the exact (molecular) nuances of hypoxia in the lung that cause pulmonary fibrosis,” Kathiriya said. “We’ve found that hypoxia is a systemic injurious factor involving a number of signaling pathways that cause fibrosis in nature, but these different pathways have common protein mediators – FAK1 and Galectin-1 – that may be leveraged as therapeutic targets for pulmonary fibrosis.”

Jaymin Kathiriya, MS, MCOM doctoral student in the Department of Pathology and Cell Biology, was among the select group of 11 students invited to give oral presentations at the 2016 USF Health Research Day.

The lab continues to work on developing a mouse model that correlates well with the slow, progressive features seen in IPF patients in the clinic, and on identifying drug candidates to test using in vivo models.

The study that second-year medical student Abby Pribish presented at Research Day was supported by a full-time summer scholarship from the Morsani College of Medicine’s Scholarly Concentrations Program. In the laboratory of Danielle Gulick at the USF Health Byrd Alzheimer’s Institute, Pribish tested the hypothesis that frequent light cycle alternations disruptive to circadian rhythm, the internal biological clock, would increase alcohol drinking in adolescent mice.

She and her lab colleagues found that breaking the circadian clock of the young mice did not cause them to drink more alcohol; however, the way the mice drank alcohol changed significantly. They consumed the same volume of alcohol in more rapid short bouts during the day.

“Messing up the light cycles caused them to binge drink,” Pribish said. “What we think is happening is that the adolescent mice seem to be intentionally spiking their blood alcohol concentration as a mechanism for coping with circadian desynchrony.”

Second-year medical student Abby Pribish was also invited to speak about her research in the oral presentation session. Her study focused on how circadian desynchrony would affect alcohol consumption in adolescent mice. -Photo by Sandra C. Roa

Pribish plans to continue to pursue her interest in addiction medicine, particularly as it pertains to adolescents — a population she says has not been studied as much as adults when it comes to alcohol use and treatment.

“Eleven percent of the alcohol consumption in the U.S. is by adolescents, and 90 percent of this (under-age) drinking is binge drinking,” she said. “It’s a huge problem… and I definitely want to be part of the solution in my career in research and as a doctor.”

Therapeutic insights from the genetics of ALS

Physician-scientist Robert H. Brown, Jr., MD, DPhil, who is nationally renowned for research on amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, delivered the Annual Roy H. Behnke Distinguished Lectureship. He is professor and chair of neurology at the University of Massachusetts Medical Center and Medical School.

Research Day keynote speaker Dr. Robert H. Brown has a record of significant discoveries in identifying gene defects that elucidate how ALS causes neurons to die.

Dr. Brown spoke about investigations by his laboratory and others to harness genetics as a pathway to understanding how ALS causes neurons to die and to gain insights into potential therapies for this progressive paralyzing neuromuscular disorder with no effective treatment. In 1993, he led a team that identified the first gene linked to hereditary ALS (copper-zinc superoxide dismutase 1, or SOD1) and mapped the mechanisms for SOD1 neurotoxicity in humans and in cellular and animal models.

Today, more than 50 ALS-related genes showing familial transmission have been discovered, and Dr. Brown’s work provides evidence that genes implicated in inherited ALS also play a role in the more common sporadic form of the disease.

The end goal is to create better models of the disease to probe therapies – everything from small molecules, biologics and antibiodies to tropic factors secreted by stem cells and gene silencing, said Dr. Brown, whose team has engineered an artificial miRNA against SOD1, which is packaged into an adenoassociated virus vector. As in cancer, multiple interventions may be required to overcome the complex patterns of gene expression in ALS, he added.

From left: Dr. Bryan Bognar, vice dean of MCOM Educational Affairs; Dr. Phillip Marty, associate vice president for USF Health Research; Dr. Paul Sanberg, senior vice president for USF Research, Innovation & Economic Development; Dr. Charles Lockwood, senior vice president for USF Health and MCOM dean; Dr. Clifton Gooch, chair of neurology; Dr. Robert H. Brown, USF Health Research Day speaker; and Dr. John Sinnott, chair of internal medicine.

“The genes involved in this disease are not what we expected, and the approaches we will need to treat the disease are yet unknown,” said Dr. Brown. “But there has been substantial progress, so I’m very optimistic that we and others will be able to work toward a treatment in the near term.”

In fact, Dr. Brown said he is he is hopeful therapies that ultimately prove useful in delaying progression and reversing symptoms in ALS may also make a difference in other degenerative brain diseases like Alzheimer’s and Parkinson’s.

In addition to Kathiriya and Pribish, the other students selected to present their research at the 7th Annual Joseph Krzanowski, PhD, USF Health Invited Oral Presentations Session were: Ngozichukwuka Agu; Faris Galambo, BS; Krishna Reddy; Alison E Roth, MPH; Stephanie Ciarlone; Jared Tur; April Lussier; Jessica M Gordon; and Rachel G. Sinkey, MD.

This year’s slate of presenters at the 26th Annual USF Health Research Day included more students and trainees and slightly fewer faculty.

Top Awards

MCOM Outstanding Postdoctoral Poster Presentation: Aurelie Joly-Amado, PhD

MCOM Outstanding Fellow Poster Presentation: Liliana Bustamante, MD

MCOM Outstanding Resident Poster Presentation: Anne Mattingly, MD (oncological sciences)

Outstanding Global Pediatric Behavioral Health Poster Presentation: Sophia Zavrou, PsyD

Outstanding Innovations in Medicine Poster Presentation: Rose Tillis

Watson Clinic Award to a Fourth-Year Medical Student: Sriram Velamuri

Dr. Christopher P. Phelps Memorial Fund Annual Morsani COM Graduate Student Travel Award: Krishna Reddy

Second-year medical student Rose Tillis explains her research findings to Joseph Krzanowski, PhD, former MCOM associate dean and professor emeritus in the Department of Pharmacology and Molecular Physiology. Tillis won an Outstanding Innovations in Medicine award for the poster titled “Hydrocolloid Bra for Nipple Sparing Mastectomy.”

Best Graduate Student Poster Presentations:

Doctoral Student Poster Presentation: Allergy and Immunology: Jillian Whelan

Doctoral Student Poster Presentation: Cancer Biology: Stephanie Buttermore

Doctoral Student Poster Presentation: Cardiovascular and Clinical Science Research: Natascha Alves

Masters Student Interdisciplinary Research: Kathryn Fomuke and Andrew McGill

Doctoral Student Poster Presentation: Molecular and Cellular Biology: Jaymin Kathiriya

Kathiriya’s presentation “Hypoxia Exacerbates Pulmonary Fibrosis via FAK1 and Galectin-1 In Vitro and In Vivo” won one of the MCOM Best Doctoral Student Poster Presentation awards. Here he is congratulated by Internal Medicine Chair Dr. John Sinnott.

Best Medical Student Poster Presentations:

Med I Student Poster Presentation: Interdisciplinary Research: Nima Hosseinian

Med II Student Poster Presentation: Interdisciplinary Research: Curtis Gravenmier

Med II Student Poster Presentation: Interdisciplinary Research: Kristen Marcet

Med II Student Poster Presentation, Case Studies and Chart Reviews: Andrew Lai, MPH, Anthony Clark, and Luis Perez-Mena

Med III Student Poster Presentation, Case Study and Chart Review: Kyle Achors

Med III Student Poster Presentation, Empirical Study: Laura Kidd

Med IV Student Poster Presentation Case Study and Chart Review: Cheryl Godcharles

Andie Dodge, a Department of Molecular Physiology and Pharmacology research technician in Dr. Edwin Weeber’s laboratory, was first author on a poster presentation titled “Knock down of Disabled-1 inhibitory neurons reveal novel role in synaptic plasticity.”  Dodge is applying for graduate school.

Best Medical Resident Poster Presentations:

MCOM Resident Poster Presentation: Interdisciplinary Case Studies: Jennifer Divine, MD, and Joanna Robles, MD

MCOM Resident Poster Presenation: Case Study and Chart Review: Karina Vivar, MD

MCOM Fellow Poster Presentation: Case Study and Chart Review: Sangeetha Prabhakaran, MD

Ruisheng Liu, MD, PhD (center), professor of molecular pharmacology and physiology, and Ingrid Bahner, PhD (right), associate professor of molecular medicine, pause from their busy judging schedule to pose for a photo. Some 83 faculty from all USF Health colleges and some affiliates served as judges for Research Day.

Subhra Mohapatra, PhD, (right) associate professor of molecular medicine, with Patricia Askins, a student from Sarasota High School who has been mentored by Dr. Mohapatra for the last year.

Keynote speaker Dr. Brown stopped by to look at the science projects exhibited by middle school students from Berkeley Preparatory School in Tampa. Kennedy Wakefield (left) and Bella Gonzalez-Portillo discussed their poster presentation “The Effects of Over-the-Counter Drugs” with him.

Students retrieve their Research Day award certificates.

Video by Sandra C. Roa and photos by Eric Younghans, USF Health Communications & Marketing

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.

***
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.

***
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.”

***
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.”

***
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.”

***
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.