HELP & HOPE
SYMPOSIUM
April 9, 2026 | Valley Forge Casino & Resort, Stardust Hotel
9:00 AM - 10:00 AM
Registration Continental Breakfast and Vendor Expo Begins
10:00 AM - 10:15 AM
Welcome Remarks by Tony Heyl and Jeff Kurkjian
10:15 AM - 11:00 AM
(choose one)
Scalable AI-Driven Solutions for Genomic Medicine Production
Dr. Irene Rombel | BioCurie; Columbia University
Genomic medicines – spanning cell, gene, and nucleic acid therapies (CGT) – have the unprecedented potential to treat and cure genetic disorders, cancer, and many other devastating diseases. However, the future of CGT is seriously hampered by very real and practical challenges in manufacturing. Today, next-gen technologies are producing a multiplicity of data that could be transformed into actionable knowledge for production – with the help of AI. BioCurie’s mechanistic AI (mAI)-driven software enables biopharma to improve speed, quality, cost of CGT production, thereby increasing patient access. Developed by Richard Braatz, thought leader in AI for biomanufacturing, the mAI BioCurie PlatformTM empowers lab scientists to conduct millions of experiments in silico to quickly predict and optimize manufacturing conditions for each therapy. The goal is to transform CGT process development and manufacturing from an inefficient, labor-intensive, and unpredictable state to a data-driven, intelligent, and robust state that enables faster delivery of lifesaving therapies that are safe, effective, and affordable.
A Multi-Disciplinary Approach to Understanding ALS
Dr. Daniela Zarnescu | Cell and Biological Systems, and Neurology, Penn State College of Medicine
There is increasing evidence of environmental factors contributing to motor neuron disease, including metals. In a first of its kind study in Pennsylvania, in collaboration with Dr. Simmons, we are collecting patient information about potential environmental exposures throughout lifetime. Biological samples including blood and nail clippings from ALS patients and controls are used to detect the presence of metals as well as identify genetic markers that together with information about potential environmental exposures are being analyzed together to determine risk of developing disease and effect on disease progression. The information gathered from ALS patients visiting the ALS clinic at the Penn State College of Medicine is being used to study the effect of specific environmental factors in fruit fly models of ALS that can provide rapid and disease relevant information about the contributions of various exposures to motor neuron disease and potentially identify novel strategies for preventing and treating ALS.
11:00 AM - 11:45 AM
Break and Vendor Exhibit
11:45 AM - 12:45 PM
Lunch and Keynote Presentation
Strength Through Collaboration,
Shana Dodge, PhD, and Esther Kane, MSN, RN-CDP, of the Association for Frontotemporal Degeneration; Zach Hardy of The Michael J. Fox Foundation for Parkinson’s Research; Dr. Kyra O’Brien in collaboration with the Alzheimer’s Association; and Steve Spaulding, MA, of ALS United Mid-Atlantic will share insights on how cross-organizational partnerships are accelerating research.
12:45 PM - 1:15 PM
Break and Vendor Exhibit
1:15 PM - 2:00 PM
(choose one)
Understanding ALS through genetics: new risk factors and new therapeutic targets
Frederick "Eric" Arnold | Washington University in St. Louis
Although only approximately 10% of ALS cases are classified as “familial,” growing evidence suggests that genetics plays an important role in the remaining 90% of “sporadic” ALS cases, which occur in individuals without a family history of disease. Rather than being driven by a single inherited mutation, sporadic ALS likely arises from the combined effects of multiple genetic variants, each contributing modestly to disease risk and acting in parallel with environmental and age-related risk factors. In this talk, I will introduce the methods used to identify new genetic risk factors for ALS and discuss how studying ALS genetics can inform our understanding of the key biological pathways that influence motor neuron survival. Importantly, with new (and improved) gene therapy approaches now becoming available, defining genetic contributors to sporadic ALS may uncover therapeutic targets relevant to the vast majority of ALS patients.
Unmuted - How AI Changes the ALS Conversation
Ed Mytych with David Bishop, Eric Brunner, and Stephen Cronin
For many people living with ALS (pALS), the loss of the ability to speak is a terrible reality. Today’s AI technology offers a profound solution: life-like digital avatars—complete with realistic digital voice reproductions—that provide patients with an alternate means of communication. This panel will explore personal experiences with these groundbreaking technologies, focusing on AI's potential to dramatically improve the quality of life for pALS through enhanced communication, deeper emotional connection, and expanded creative outlets.
2:00 PM - 2: 15 PM
Break and Vendor Exhibit
2:15 PM - 3:00 PM
(choose one)
Synchron BCI – Building Brain-Computer Interfaces Informed by Lived Experience
Charles Poeppelman, MS, CCC-SLP | Synchron
Brain-computer interfaces (BCIs) are moving from research labs toward clinical reality. This session introduces Synchron’s approach to developing implantable BCI technology through continuous partnership with patients, caregivers, clinicians, and advocacy communities. Rather than designing technology in isolation, Synchron integrates lived experience to guide usability, clinical workflow integration, and to help us to understand what is meaningful to patients and their families. Attendees will gain an overview of the BCI field, current clinical progress, and how the ALS community can help shape responsible innovation that prioritizes what is most meaningful and critical to people living with paralysis and those that care and support them.
Your Voice is Your Power: How Thomas Carroll’s Battle Created the Framework for Tom's Friends of ALS Bill of Rights
Tara McKean, John Russo, and Lura Sawyer
Thomas Carroll’s battle with ALS transformed his daughter, Tara McKean, from a veteran educator into a tenacious advocate. After witnessing her father suffer through medication barriers—forced to "fail" on pills he could not swallow—and life-threatening equipment delays, Tara channeled her grief into action. With a mission rooted in faith and strength, Tara drafted the "Tom’s Friends of ALS Bill of Rights." This framework demands prior authorization reform and specialized ER protocols to prevent the crisis her father faced. Today, Tara finds healing in educating families on how to advocate through the ALS United Mid-Atlantic Help & Hope Symposium. By sharing her father’s story, she ensures his light leads the way toward a system that guarantees every patient immediate, life-sustaining care and dignity.