On October 27, 2023, ALS Therapy Development Institute (ALS TDI) scientists and guest presenters from across the ALS space gathered at the ALS TDI Summit in Boston, MA, to provide the community with updates on research from the past year. They were joined by an audience from across the ALS community, including people with ALS, families, advocates, researchers, pharmaceutical industry professionals, and many more who streamed the event online.
Here is a recap of what was shared at this year’s ALS TDI Summit!
Research Updates from ALS TDI
Collaborative Science in Service of Ending ALS
To begin, ALS TDI CEO and Chief Scientific Officer Dr. Fernando Vieira delivered a presentation that focused on the day’s overall theme – how collaboration helps push ALS research forward. This included an explanation of the internal teams at ALS TDI and how they work together to power a comprehensive research program that encompasses all areas of preclinical drug development. He also described how ALS TDI partners with outside organizations to learn more about the underlying biology and possible causes of ALS, test potential treatments in cellular and animal models of ALS, and share comprehensive data about the disease
View the recording here.
ALS Research Collaborative: Tackling ALS Together
Next, Alan Premasiri, ALS TDI’s Senior Manager of Clinical Operations, gave an overview of the ALS Research Collaborative (ARC), a global initiative launched in 2023 by ALS TDI to gather and share comprehensive data from people with ALS. Alan explained how ARC evolved from ALS TDI’s Precision Medicine Program (PMP), the longest running natural history study in ALS. He detailed how the ARC Study will continue to expand data collection efforts, allowing anyone with ALS and asymptomatic gene carriers to contribute to research remotely from anywhere in the world. Additionally, he shared how these data is made available to researchers across the ALS space through the ARC Data Commons – an online data-sharing and analysis platform developed by ALS TDI – to foster collaboration and help move ALS research forward.
View the recording here.
Collaborative Approaches for Studying and Treating ALS via the Use of Animal Models
Alan was followed by ALS TDI’s Director of Pharmacology Dr. Theo Hatzipetros, who elaborated on how we work with other organizations to develop and test drugs in animal models of ALS. He described our work with six different organizations, including academic researchers, biotech companies, and other nonprofits. These projects included work to develop and refine new ALS mouse models and test a number of potential treatments in our lab. He also discussed why ALS TDI’s unique model as a nonprofit biotech makes us an appealing partner for other institutions working to develop ALS treatments.
View the recording here.
iPS Cell Biology Models and Techniques for ALS Discovery
Next, Dr. Kyle Denton, ALS TDI’s Director of Cell Biology, emphasized how contributions from people with ALS are essential to ALS TDI’s research efforts. Dr. Denton described how his team uses induced Pluripotent Stem Cell (iPSC) models of ALS, some of which are sourced from participants in the ARC Study, to learn more about the disease and test potential drugs. He shared examples of how these models have been used in experiments to help advance our understanding of topics such as C9orf72-realted genetic ALS and TDP-43 pathology in ALS.
View Kyle’s recording here.
An Innovative Tool for Tailored Clinical Trial Recommendations
ALS TDI’s Director of Community Outreach and Engagement Dr. Nadia Sethi then took the podium to share an innovative platform developed by ALS TDI to help people with the disease learn more about clinical trials. She demonstrated how a guided questionnaire helps users to learn about research opportunities, set personal priorities, and identify trials that meet their specific criteria. In addition, users can create a personalized account to save trials, make notes, and share comments. She also detailed how the creation of this tool was a collaborative effort with the ALS community, involving contributions from the ALS TDI team, ALS advocates, and people with ALS. The tool will be made available on ALS TDI’s website in early 2024.
View Nadia’s recording here.
ALS TDI Scientist Panel
Following the scientist presentations, Dr. Sethi hosted a panel which included four of ALS TDI science team members:
- Shelby Ellis, Science Intern II
- Josh Kidd, Research Associate III
- Anna Gill, Associate Scientist III, Program Manager
- Therese Dane, Associate Scientist III
The panelists answered a series of questions including what brought them to ALS TDI, what excites them about their current work, and what makes ALS TDI such a unique place.
View the panel recording here.
After breaking for lunch, the summit reconvened for series guest presentations from other leaders in the ALS research and drug development space.
A Veteran Physician-Scientist Reflects on Advances in ALS: From Early Biotech to the Present
First was Dr. James Larrick, Co-Founder and Managing Partner of Corsalex, a company working to develop C9orf72-related ALS and FTD. Dr. Larrick’s presentation covered the progress of drug development research over his many decades in the biotech industry. He described how, over the past 50 years, technologies for understanding and treating disease have grown in ways that would have once seemed impossible or inconceivable to most researchers in the field. He covered how this progress has revolutionized the treatment of diseases like cancer in this time, and how this could provide a roadmap for a similar revolution in ALS.
View Dr. Larrick’s recording here.
The Development of Tofersen for SOD1-ALS
Dr. Larrick was followed by Dr. Stephanie Fradette, Senior Medical Director for Clinical Development at Biogen. Dr. Fradette described her experience as the clinical development lead for Qalsody, also known as tofersen, a drug for SOD1-realted ALS that received FDA approval earlier this year. She described the development of the treatment, from the discovery of SOD1, to preclinical testing, to trials, and finally to reaching approval. She also emphasized the importance of research into neurofilament light chain (NFL), an ALS biomarker, in designing, analyzing, and ultimately winning approval through the clinical trials for the treatment.
View Dr. Fradette’s recording here.
The Future of ALS Research: Collaboration Fueling Drug Development
For the last science presentation of the day, Dr. James Berry, Associate Chief of Neuro-therapeutics and Chief of the Division of ALS and Motor Neuron Diseases, delivered a presentation about collaboration across the ALS research field. He emphasized how collaboration between researchers and institutions is essential in solving the problem of ALS, and how people in the industry are working together. His presentation touched on how programs and organizations such as the Northeast ALS Consortium (NEALS), Answer ALS, the Healey Platform Trial, and ALS TDI’s ARC Data Commons, are fostering collaborations between scientists to share knowledge to better our understanding and develop treatments for the disease.
View Dr. Berry’s recording here.
Honoring the ALS Community
Following the day’s scientific presentations, ALS TDI’s Vice President of Development Carol Hamilton took the podium to announce this year’s Leadership Awards. These annual awards honor members of the ALS community who have gone above and beyond to support research and advocate for the needs of people with the disease.
This year’s Leadership Award winners included:
- Alexander Heywood Award – Clara & Ellie Wetzel and Brady & Brooklyn Yozwiak, in recognition of these two young pairs of siblings’ extraordinary efforts to raise funds and awareness for ALS research.
- Stephen Milne Adventurous Spirit Award — Brooke Eby & Juan and Meg Reyes, in recognition of the inspiration both have provided to the ALS community and beyond. Brooke has touched many thousands of people through her funny and relatable videos about living with ALS as a young person, while raising hundreds of thousands of dollars, on social media. Juan and his wife Meg, in addition to their advocacy efforts, inspired many by embarking on and documenting a “bucket-list” RV trip around the United States.
- Mary Lou Krauseneck Courage & Love Award — Lorri Carrey, to honor her resilience over nearly two decades of living with ALS, and her work spreading awaren=ss and sharing inspiration with her podcast, I’m Dying to Tell You.
- Fran Delaney Challenge & Respect Award — Mandi Bailey, to honor her tireless mission to advocate for the needs of veterans with ALS and raise money for ALS research.
View the recording of the Leadership Awards presenation here.
ALS TDI hopes that the ALS Summit provided valuable information and helped to inform the community about our current work.
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