The annual ALS TDI Summit, hosted by the ALS Therapy Development Institute (ALS TDI), offers the ALS community a unique opportunity to hear firsthand about the latest developments in ALS research.

This year’s Summit, taking place on October 9, will highlight research focused on both sporadic and familial forms of ALS. The event will feature presentations from ALS TDI scientists, a panel discussion with researchers and community members, and talks from guest experts across the field.

ALS TDI Research Presentations

Join us as the Summit opens with key ALS TDI research updates from:

  • Fernando Vieira, MD, CEO & Chief Scientific Officer, ALS TDI; Building What ALS Demands
  • Kyle Denton, PhD, Director, Cell Biology, ALS TDI; Modeling TDP-43 Loss-of-Function in Human Motor Neurons: From Cryptic Splicing to Drug Discovery
  • Theo Hatzipetros, PhD, Senior Director, Pharmacology, ALS TDI; Leveraging the SOD1 Mouse Model to Evaluate Diverse Therapeutic Mechanisms in ALS
  • Alan Premasiri, Director, Clinical Operations, ALS TDI; Expanding Capabilities of the ARC Platform 
  • Danielle Boyce, MPH, DPA, Director, Data Science and Biomedical Informatics, Principal Investigator, Real World Evidence, ALS TDI; Faster with AI, Guided by People: Data Science on the ALS Clock

ALS TDI Summit Panel

Hereditary vs. Sporadic ALS: What Have We Learned and Where Are We Going?
Join a panel of leaders in the ALS space for a discussion about how the latest innovations in ALS research are being applied across both hereditary and sporadic ALS, and how discoveries in one form of the disease can inform treatment strategies for others. 

Moderated by: Yentli Soto Albrecht, PhD

Panelists Include:

  • Fernando Vieira, MD, CEO/CSO, ALS TDI
  • Michael Robinson, MD, living with ALS
  • Steven Altshuler, PhD, Professor, Pharmaceutical Chemistry, University of California, San Francisco
  • Danielle Boyce, MPH, DPA, Director of Data Science, ALS TDI

ALS TDI Summit Guest Speakers

The afternoon will include guest presentations from a variety of industry leaders working across the ALS research space. The afternoon session will be divided into two sections covering innovations in Cell Biology and Basic Research, and the use of digital measures as Clinical Endpoints

Cell Biology and Basic Research Presentations

Lani Wu, PhD

Professor, Pharmaceutical Chemistry, University of California, San Francisco

From Skin Biopsy to ALS Subtype: A Phenotypic Platform for Patient Stratification and Therapeutic Discovery
Dr. Wu will discuss research to develop a scalable platform that combines transcriptomic profiling and high-content imaging of patient fibroblasts to identify phenotypes associated with genetically defined ALS subtypes, including FUS and SOD1. By using these genetic forms as biological anchors, Dr. Wu and her colleagues aim to detect related signatures in sporadic ALS and establish a framework for patient stratification and therapeutic discovery.

David Shechter, PhD

Professor of Biochemistry, Albert Einstein College of Medicine

PRMTs in ALS: From RNA Regulation to Therapeutic Opportunity 
PRMT (Protein Arginine Methyltransferase) enzymes modify many of the proteins that control RNA processing and organization - functions that repeatedly fail in ALS. Dr. Shecter will discuss why PRMTs may offer a therapeutic target, using C9orf72-derived poly-GR toxicity and our emerging work on RNA binding, protein interactions, and nucleolar organization as a mechanistic case study.

Clinical Endpoints Presentations

Marcin Straczykiewicz, PhD 

Research Consultant for Digital Health Technologies

Measuring Change in Everyday Life with ALS
Dr. Strączykiewicz is an independent consultant whose research utilizes wearable activity monitors to track changes in movement and daily activity over time in people with ALS. He will discuss his current work to investigate the potential of these measures to help capture changes that may not be apparent during clinic visits alone.

Smith Heavner, PhD, RN, FCCM

Senior Scientific Director, Data Collaboration Center, Critical Path Institute

Fit-for-Purpose Is Not One-Size-Fits-All: From Digital Measures to Regulatory Impact in ALS
Digital measures are promising tools in ALS research, but a measure that works well for one purpose or group of people may not be appropriate for another, particularly in a heterogeneous disease like ALS. Dr. Heavner will discuss how clearly defining what we want to measure, in whom, and why helps determine what evidence is needed before that measure can inform regulatory decisions. He will also cover ways that patients, researchers, technology developers, and regulators can engage early in development to help digital measures address meaningful needs and ultimately support regulatory decision-making.

Register Now to Attend the ALS TDI Summit!

Registration for both online and in-person attendees is free, but please note that registration is required to attend. For more information about attending the ALS TDI Summit, visit https://www.als.net/summit

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