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

This week’s New England Journal of Medicine reported results of the pivotal CENTAUR clinical trial; the USF Health ALS Clinic was one of only two sites in Florida for the study

Tampa, FL (Sept. 2, 2020) – Despite two approved medications and at least 100 clinical trials later, an urgent need for new treatments to improve the lives of patients with amyotrophic lateral sclerosis (ALS), a relentlessly progressive and fatal disease, continues to exist.

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This week’s New England Journal of Medicine published results of the highly anticipated CENTAUR clinical trial, and included among the study’s coinvestigators was Tuan Vu, MD, professor of neurology at the Morsani College of Medicine and director of the USF Health ALS Clinic.

CENTAUR was a 24-week randomized, double-blind trial Phase 2/3 trial of 137 adults with ALS conducted across 25 top medical centers in the U.S. through the Northeast ALS (NEALS) Consortium. USF Health was one of two Florida sites to evaluate the safety and effect on disease progression of Amylx Pharmaceuticals’ investigational neuroprotective therapy, known as AMX0035. The oral therapy combines two different medicines: sodium phenylbutyrate and taurursodiol.

Using the Revised ALS Functional Rating Scale (ALSFRS-R) to measure daily functions such as the ability to walk, dress independently, self-feed, speak and breathe, the CENTAUR investigators demonstrated that treatment with AMX0035 decreased the rate of decline in patients with ALS compared to those with ALS receiving a placebo. The therapy was generally well tolerated, with similar rates of adverse events reported in the AMX0035 and placebo groups. For more details on the CENTAUR trial, including the open label extension study, and next steps, click here. 

“ALS is likely the end result of several different pathological processes leading to the death of nerve cells in the brain and spinal cord that control voluntary muscle movements — so there is no one treatment solution. To combat this complex neurodegenerative disease we need to target more than one process at the same time,” Dr. Vu said.  “This investigational drug protects motor neurons in a different way than the two currently approved ALS medications (riluzole and edaravone). It was beneficial in slowing disease progression, allowing patients to remain functional for a longer period of time.”

Only two drugs to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, have been approved by the FDA over the last 25 years.

“CENTAUR met its prespecified primary outcome, showing a clinically meaningful and statistically significant treatment benefit on the ALSFRS-R, the most commonly used scale in clinics worldwide to measure function in ALS,” said Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General and assistant professor of physical medicine and rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital. “These results represent a major milestone for the ALS community, and I am thrilled about the promise of this therapy for people with ALS.”

AMX0035 was the first investigational therapy to demonstrate statistically significant benefit on the prespecified primary outcome in patients with ALS since edaravone, an intravenous drug approved by the U.S. Food and Drug Administration (FDA) in 2017.  The first and only other commercially available drug to treat ALS, riluzole, was FDA approved 25 years ago.

The NEJM study authors concluded that sodium phenybutyrate/taurursodiol (AMX0035) resulted in slower progression of ALS over 24 weeks as measured by the ALSFRS-R total score, but recommended longer and larger trials to further evaluate the therapy’s effectiveness and safety.

Dr. Tuan Vu, professor of neurology, directs the USF Health ALS Clinic. Below: Dr. Vu performs an electromyography (EMG), one of the tests used to help diagnose ALS.

Zbigniew (Ziggy) Grajzer of Clearwater, Fla, was diagnosed with ALS in November 2019 and began treatment at the USF Health ALS Clinic in February 2020. He takes both edaravone and riluzole and remains hopeful about any steps forward to find new ALS treatments to improve quality of life and maintain independence.  While he was not enrolled in CENTAUR, Grajzer, 69, participates in other ALS clinical trials at the USF Health whenever he can. “It is the only way to find a cure,” he said, “if not in time for me, then to help others in the future.”

Grajzer said he feels fortunate that his ALS appears to be progressing slowly, but acknowledges the struggles of living with a disease that gradually robs an individual’s ability to control muscle movement. He has lost use of his left hand, his legs are weaker, and he tires easily, even walking from one room to another.  “I enjoyed going everywhere, to the beach and the mountains. Now most of what I do is around the house. But at least I can still move, speak, eat, breathe and think, and I’m grateful for that,” he said.

Zbigniew (Ziggy) Grajzer began treatment at the USF Health ALS Clinic earlier this year.

CENTAUR, the recipient of an ALS Accelerated Therapeutics grant, is supported by The ALS Association, ALS Finding a Cure, a program of The Leandro P. Rizzuto Foundation, the Northeast ALS Consortium, Healey Center for ALS at Mass General, and was funded in part by the ALS Ice Bucket Challenge.

ABOUT USF HEALTH ALS CLINIC
The USF Health Amyotrophic Lateral Sclerosis (ALS) Clinic in Tampa, Fla., offers the latest treatment, clinical research opportunities, supportive care and educational resources to help patients, their families and caregivers manage the effects of ALS. The clinic has been recognized by The ALS Association as a Certified Treatment Center of Excellence.

About AMYOTROPHIC LATERAL SCLEROSIS (ALS)
ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. The vast majority of patients with ALS (>90%) have sporadic disease, showing no clear family history. Approximately 6000 people are diagnosed with ALS in the United States every year with an approximately similar number of deaths every year.

ABOUT AMX0035
AMX0035 is an investigational neuroprotective therapy designed to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

-Video by Allison Long, USF Health Communications and Marketing

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.

Sadie, a rescue dog from Project PUP, makes friends with Matt June, a patient of the USF ALS Center.

Patients visiting the USF ALS Center on April 5 were greeted with a wagging tail and soulful eyes begging for attention.

Sadie, a senior rescue dog from PROJECT PUP (Pets Uplifting People), was on hand to spend some time with patients while they waited and even during their clinical time with doctors and staff at the multidisciplinary clinic.

The Center plans to make Sadie, and possibly other canine friends, part of a pet therapy program at its monthly clinic housed within the USF Health Morsani Center for Advanced Healthcare, said Brittany Harvey, staff assistant and research support specialist for the ALS Center.

Sadie’s owner Christine Hamacher, a volunteer with Project PUP, said research has shown that interaction with good-tempered pets like Sadie can help calm the anxiety of patients and their families, who spend quite a bit of time being assessed by healthcare team members during their clinic appointments. “Just having her (Sadie) in the same room helps them to speak more comfortably and freely with doctors and staff,” Hamacher said.

Sadie may not know she’s doing all that.  Waiting in an exam room with patient Matt June and his wife Jackie, she appeared more than content to solicit hugs, get scratched behind her silky ears and dole out doggie kisses.

Matt and wife Jackie June with Sadie

The USF ALS Center, directed by Dr. Tuan Vu, opened in October 2010 as the first multidisciplinary university clinic of its kind in Tampa Bay and all of Central and West Florida.

About 200 patients a year visit the clinic, which is dedicated to developing new and effective therapies for ALS, or amyotrophic lateral sclerosis. Also, known as Lou Gehrig’s disease, the neurodegenerative disease progressively paralyzes the individual, attacking nerve cells and pathways in the brain and spinal cord.  The USF ALS Center belongs to the world’s largest ALS research network, the Northeastern ALS Alliance.

Photos by Eric Younghans, USF Health Communications