How one Virginia hospital is icing out ALS
A decade after the first ice bucket challenge, the VCU Health ALS Clinic is ramping up research diagnosis and treatment on the national stage.
September 12, 2024
Marking ten years since the start of the challenge, members of the VCU Health ALS Clinic continue the tradition of dumping buckets filled with ice and water over their heads to raise awareness about ALS (Sara McCloskey, Enterprise Marketing and Communications).
By Leigh Farmer
The ALS Bucket Challenge is 10 years old.
But the resolve to study and bring awareness to the illness hasn’t aged a day.
Look no further than the buckets of ice-cold water people are still dumping on their heads, live on social media.
That includes the team at the VCU Health ALS Clinic. They recently gathered outside of VCU Health’s Short Pump Pavilion with ice buckets and a challenge for every other clinic in Virginia. In no uncertain terms – they are ready to keep it all going.
But there is nothing cold about this team.
They are on fire.
The VCU Health ALS Clinic has tripled in size since its inception in the summer of 2011. Designated as an ALS Association Center of Excellence, it is the largest clinic in the commonwealth.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive disease that slowly “turns off” the motor neurons in the brain and spinal cord. This eventually leaves a person unable to speak, eat, move, and breathe. It progresses quickly. Typically, once diagnosed, a person lives between two to five years.
While there is no known cure, the VCU Health ALS Clinic is part of several major clinical research studies. It is one of 34 sites in the National Institutes of Health’s ALL ALS Consortium, as well as a site for the Federal Drug and Food Administration’s Natural History Consortium.
“We went from having a very small research footprint to being a destination research program for the entire region,” said Kelly Gwathmey, M.D., director of the clinic.
(Right) Kelly Gwathmey, M.D., is the director of the VCU Health ALS Clinic (Sara McCloskey, Enterprise Marketing and Communications).
Since Gwathmey’s arrival in 2019, the ALS clinic has morphed in size, national standing, and capacity for “game changing” research. Just four years ago, there was only one research coordinator for the entire neuromuscular division. Now, there are four researchers dedicated to studying this illness.
At the VCU Health ALS Clinic, specialists see one to three new patients every week. Annually, they provide care to approximately 200 patients. That is a lot for a disease still described as “rare.” Barring a family history of ALS, the chance of developing it is less than 1%. Roughly, 30,000 people are living with the disease in the U.S. An estimated 5,000 new cases are diagnosed each year.
Gwathmey spoke with VCU Health News about her team’s multi-disciplinary approach and how the diagnosis, treatment, and research of this disease has progressed since the 2014 bucket challenge initiative began.
Three young men living with ALS began this ice bucket phenomenon ten years ago with the intention of raising awareness and money for research. Since that time, from a research standpoint, have you seen significant changes? What’s the most impressive change you’ve seen?
Since the original challenge, more than $100 million has been raised to further research and to support the care of ALS patients. The challenge also raised awareness, bringing ALS into the spotlight and making it a household name.
When it comes to medication, before the ice bucket challenge initiative, there was only one FDA-approved medication. Now there are three. One of those medications, tofersen, represents a remarkable breakthrough as the first targeted therapy for a form ALS that is caused by an inherited genetic mutation (SOD 1). Though this form of ALS is rare, some patients treated with this drug are seeing their symptoms stabilize or get better.
Our lawmakers are taking notice, as well. Legislators at the state and federal levels have increased funding for research, resulting in the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) which was signed into law on December 23, 2021, by President Joe Biden.
From an awareness standpoint, what changes have you seen?
Prior to the ice bucket challenge, very few people were truly aware of ALS. It was a disease that most people associated with Lou Gehrig or Stephen Hawking - high-profile figures diagnosed with the disease. There was no personal connection.
Now, thanks to the challenge, communities enthusiastically rally behind our ALS patients. ALS walks and fundraisers to support research and ALS clinics happen frequently. This is wonderful because our need to find better ALS treatments and continue to improve patient care and access to care are now commonly discussed topics.
What are the components of a solid care plan for a patient diagnosed with ALS?
I often tell our patients that there are three main pillars to the care plan at the VCU Health ALS Clinic. The first pillar is multidisciplinary care – the standard care model for our patients. We know that this type of care results in improved life expectancy and quality of life.
Our patients receive access to a nurse navigator, occupational therapist, registered dietician, respiratory therapist, speech language pathologist, genetic counselor, licensed clinical psychologist, and clinical research coordinators. This is in addition to the neurologist who will monitor you closely.
The second pillar is providing access to both disease-modifying therapies, which slow disease progression, and symptomatic treatments. Symptomatic treatments address common symptoms such as cramps, constipation, mood disturbances, and excessive saliva.
The last pillar involves providing our patients access to clinical research, regardless of the state of a person’s disease. This includes natural history studies and investigational drug trials.
The VCU Health ALS Clinic cares for about 200 patients annually (Sara McCloskey, Enterprise Marketing and Communications).
What can we expect in the next ten years from the VCU Health ALS Clinic team?
The clinical research programs will continue to expand. Over the past five years, our clinical trial portfolio has grown tremendously. Beyond the NIH and FDA funded consortiums, VCU Health is also a Northeast ALS Consortium (NEALS) site and Healey Platform site. This robust involvement in ALS research at a national and international level has elevated the reputation of the clinic and allowed us to expand our research offerings for our patients. Our ten-year plan also includes recruiting an ALS translational scientist who can assist in bringing promising therapeutics from “bench to bedside.”
The VCU Health ALS Clinic has been piloting a Rapid Access Clinic since September 2022. It was designed to shorten the delay to diagnosis for our ALS patients by raising disease awareness and providing an urgent appointment for any patient suspected to have ALS. This program helps a patient bypass the traditional referral route - which can delay a diagnosis for months. Over the next several years we plan to expand this program to patients across the commonwealth of Virginia.
How long will your team be pouring ice buckets of water on your heads?
Our team will continue to pour buckets of ice water on our heads until a cure is discovered, and ALS is a distant memory. Until this occurs, our clinical and research teams will continue to grow and expand to meet the needs of our patients, their families and caregivers, and our community. Support of ALS research and clinical care, through the ice bucket challenge and other efforts, is necessary to continue to move this field forward and the VCU Health team is proud to be at the forefront of these important initiatives.
