On Thursday, April 10, ALS United Mid-Atlantic hosted the Help & Hope Symposium at the Valley Forge Casino & Resort, Stardust Hotel. More than one hundred attendees gathered to hear the latest information on ALS care and research. Attendees included individuals with ALS, caregivers, clinicians, and those interested in learning more about advances in research.
The event featured keynote speaker, Dr. Zachary Simmons, who served as site principal investigator for more than 30 clinical treatment trials and has published extensively on quality of life, ethics of care, and end-of-life issues. The Penn State ALS Center pioneered the use of telemedicine and remote monitoring in ALS. He was awarded the 2024 Forbes Norris Award by the International Alliance of ALS/MND Associations for his clinical and research contributions to ALS. Dr. Simmons currently serves as Editor-in-Chief of Muscle & Nerve.
In addition to the keynote presentation, the symposium featured speakers from ALS United Mid-Atlantic, ALS Therapy Development Institute (ALS TDI), The Association for Frontotemporal Degeneration, HDC MidAtlantic, Lehigh Valley Health Network, and Thomas Jefferson University Jefferson Weinberg ALS Center.
“We are excited by the turnout for this year’s Help & Hope Symposium, which featured more speakers than ever before and a comprehensive vendor exhibit, allowing individuals with ALS to discover a wider range of resources. It was fantastic to see a record turnout of people with ALS and their caregivers. Our goal was to provide a variety of topics to educate the ALS community,” said Kerry Braun, Director of Community Engagement.
The event was proudly sponsored by The Association for Frontotemporal Degeneration, Fidelity Home Care of PA, Placing Stones Studio, Lehigh Valley Fleming Neuroscience Institute, and National Seating & Mobility.
Presentation Summaries
Recordings of their presentations are available on our YouTube account @ALSMidatlantic.
ALS and Research
Alissa Romano, DO | Lehigh Valley Health Network
This presentation described clinical research and the different ways patients with ALS can get involved with research. Dr. Romano also briefly discussed research at the Lehigh Valley Health Network.
ALS Multidisciplinary Clinic: Improving a Successful Model of Care
Zachary Simmons, MD | ALS Clinic and Research Center, Penn State Health Milton S. Hershey Medical Center
The ALS Multidisciplinary Clinic has been the model for ALS care for more than 40 years. It prolongs survival and results in better overall quality of life, as perceived by people with ALS, than traditional single-physician care. However, even successful models can be improved upon.
The presentation discussed a number of initiatives that are aimed at such improvement. These include: more effective communication of clinic recommendations; better use of telehealth, facilitation of communication between persons with ALS and their healthcare team between clinic visits, home monitoring of physical function between clinic visits, improved knowledge about timing of ventilatory and nutritional support, more attention to quality of life as perceived by persons with ALS, increased attention to cognitive and behavioral impairment, optimization of discussions of goals of care and end-of-life care, and better integration of research into clinic visits.
Bridging Disease Mechanisms and Therapeutic Discovery: Identifying Candidate Target Molecules for Genetic and Sporadic ALS
Brigid Jensen, PhD | Thomas Jefferson University, Department of Neuroscience, Jefferson Weinberg ALS Center
Brigid’s laboratory uses mechanistic understanding from wet lab research modeling forms of ALS to drive potential therapeutics, as well as patient-derived models to identify biomarkers and key targets for future investigation.
In the presentation she discussed a story in each of these areas, highlighting the work of her laboratory and ongoing collaborative work in the Jefferson Weinberg ALS Center. In the first she described how cellular studies, and a novel in vivo animal model have been used to identify how normally supportive glial cells instead drive neuron cell death in genetic FUS-ALS, and how a specific target molecule with therapeutic potential for this sub-population of patients has been identified. In the second vignette, she explained how patient-derived biofluids, skin cells, and postmortem tissue are being harnessed as a tool for identifying biomarkers and therapeutic targets for sporadic ALS.
FTD and ALS; When Two Paths Meet
Kim Jenny, MS, LCGC | The Association for Frontotemporal Degeneration
Kim reviewed the basic principles of genetics and the intersection between FTD and ALS. Previously, ALS was thought to be purely a movement disorder, while FTD was considered purely a cognitive or behavioral form of dementia. The shared genetics between FTD and ALS means families may experience both FTD and ALS. Understanding the connection between FTD and ALS is an area of active research, and our knowledge of the underlying genetics, pathology, and clinical features are increasingly relevant in efforts to develop treatments for everyone impacted by FTD and ALS.
Home Accessibility Options for People with ALS / / Accessibility and Affordability
Alisa Brownlee, ATP, CLIPP, CAPS, WPS, CEAC/Mastery | ALS United Mid-Atlantic
Kimberly Krauter | HDC MidAtlantic
Most homes are not designed or constructed to accommodate the needs of disabled people in terms of accessibility, safety, independence, and location. Adapting a living environment to meet the changing needs of ALS may become a necessity as these diseases progress. However, due to progressive disease changes, typical structural home modifications are often not appropriate. The cost, time, and daily upheaval of having structural modifications done to the home increase the emotional and financial burden of living with ALS and other diseases.
Accessing guidance from a person trained in home accessibility and safety can help families to identify potential physical barriers to key daily functions. The range of possible modifications supports safety, decreases harms and maximizes quality of life as determined by those diagnosed and their caregivers.
Social Worker Role Within the ALS Community
Anne Cooney, MSW, LSW and Melissa Coll, MSW, LSW | ALS United Mid-Atlantic
Anne and Melissa’s program educated attendees about the role of the ALS social worker. The two shared more details about the impact the social worker has on patients and families within the ALS Community. At the end of the program, they discussed various services and programs offered by ALS United Mid-Atlantic to support people living with ALS and their families.
Updates From the Lab, and How ALS Families Are Engaging with ALS TDI
Carol Hamilton | ALS Therapy Development Institute (ALS TDI)
The ALS Therapy Development Institute is the world’s largest independent ALS research institute, focused solely on ending ALS. During Carol’s presentation, she discussed two drugs that have come through ALS TDI are now in clinical development, and two current programs in the lab are advancing toward IND-enabling (the work required by the FDA before a drug can be tested in humans).