News & Stories
ALS Network Research Summit 2026: Post‑Summit Highlights
Three Days of Science, Translation, and Momentum
The ALS Network Research Summit convened global leaders across academia, industry, and clinical care to accelerate progress toward effective treatments and cures. Across biomarker discovery, gene therapy, advanced disease models, precision medicine, and clinical translation, the Summit highlighted how multidisciplinary collaboration is reshaping the ALS research landscape.
Opening & Vision Setting
Clive Svendsen, PhD (Cedars‑Sinai), Martina Wiedau, MD (UCLA), Leslie Thompson, PhD (UC Irvine), and Sheri Strahl, MPH, MBA (ALS Network) opened the Summit by underscoring the urgency of translational science, the importance of collaboration across sectors, and ALS Network’s role as a catalytic funder supporting bold, high‑impact ideas. Their remarks set the tone for a meeting focused on rigor, innovation, and patient‑centered outcomes.
Opening Plenary: Proteomic Biomarkers for ALS
The Summit launched with a strong call to action: ALS can—and must—be measured more precisely. Bryan Traynor, MD, PhD (NIH) delivered a powerful opening plenary on integrating genomics, transcriptomics, and proteomics to identify biomarkers that improve ALS diagnosis, prognosis, and patient stratification. His work highlighted how systems biology can directly inform precision clinical trials and therapeutic development. This talk underscored a central theme echoed throughout the meeting: better measurement unlocks better medicine.
Session I: ALS Biomarkers — Bridging Research and Clinical Practice
Chair: Bob Bowser, PhD (Barrow Neurological Institute)
This session focused on translating biomarker discovery into tools that matter for patients and clinicians. Jeff Rosenfeld, MD, PhD (Loma Linda University) emphasized what clinicians and patients need from biomarkers in real‑world care. Alex Sette, Dr. Biol. Sci. (La Jolla Institute for Immunology) explored autoimmune responses to C9orf72‑derived peptides, expanding understanding of immune involvement in ALS. Noah Zaitlen, PhD (UCLA) showcased the promise of cell‑free DNA epigenetic biomarkers for non‑invasive disease monitoring and longitudinal tracking. From immune signatures and epigenetic signals to clinician‑defined needs, the discussion highlighted a field moving decisively toward biomarker‑informed diagnosis, care, and clinical trials.
Session II: What Can ALS Learn from Other Neurodegenerative Disorders?
Chair: Steve Finkbeiner, MD, PhD (Gladstone Institutes / UCSF)
Speakers highlighted shared disease mechanisms across neurodegeneration. Leslie Thompson, PhD (UC Irvine) described unexpected overlap in TDP‑43 pathology between ALS and Huntington’s disease. Dan Child, MD, PhD (University of Washington) examined age‑related TDP‑43 pathology (LATE) and its frequent co‑occurrence with ALS. Jeff Rothstein, MD, PhD and Alyssa Coyne, PhD (Johns Hopkins University) detailed nuclear pore and transport dysfunction as central drivers of sporadic ALS. By looking beyond ALS, this session revealed striking convergence across neurodegenerative disorders. Shared TDP‑43 pathology, age‑related co‑pathologies, and nuclear transport defects emerged as unifying threads. These insights are reshaping how ALS is understood—not as an isolated disease, but as part of a broader neurodegenerative landscape ripe for cross‑disease learning and therapeutic reuse.
Session III: Industry Updates I — Therapeutic Innovation in Action
Industry leaders shared translational progress from bench to bedside. Justin Ichida, PhD and Wen‑Hsuan Chang, PhD (AcuraStem) presented development of SYF2 antisense oligonucleotide therapies. Matthew MacDougall, MD (Neuralink) highlighted brain‑computer interface trials restoring communication and device control for people with advanced ALS. Mike McGrath, MD, PhD (Neuvivo, Inc.) discussed NP001 and innate immune modulation in slow progressing ALS, while Peter Vanderklish, PhD (Spinogenix) shared Phase 2a clinical trial results for Tazbentetol targeting synaptic dysfunction.
Session IV: Gene Therapy Frontiers
Chair: Martina Wiedau, MD (UCLA)
This session showcased next‑generation gene‑based strategies. Laura Ferraiuolo, PhD (Insmed) discussed non‑cell autonomous mechanisms and RNA metabolism in ALS. Gene Yeo, PhD, MBA (UC San Diego) highlighted RNA‑targeting therapeutic approaches, and Beverly Davidson, PhD (Children’s Hospital of Philadelphia) presented advances in viral vector and CNS‑targeted gene therapy platforms. This session underscored how rapidly gene-based therapies are moving from concept to clinic, with advances in RNA-targeting strategies and CNS-directed delivery platforms expanding the range of treatable ALS mechanisms. Together, these approaches signal a future where precision gene therapies can address both cell-intrinsic and non-cell autonomous drivers of disease, opening new and potentially transformative therapeutic pathways.
Session V: Advanced Models of ALS
Chair: Gene Yeo, PhD, MBA (UC San Diego)
Speakers demonstrated how human‑relevant models are transforming ALS research. Andras Lakatos, MD, PhD (University of Cambridge) introduced next‑generation corticospinal‑myosphere organoids. Clive Svendsen, PhD (Cedars‑Sinai) presented organ‑on‑chip systems and single‑cell technologies, while Layla Starr, PhD (Synapticure) described 3D brain spheroids for disease modeling and therapeutic screening.
Session VI: Precision Medicine Approaches
Chair: Leslie Thompson, PhD (UC Irvine)
Precision medicine took center stage with Neil Shneider, MD, PhD (Columbia University / Silence ALS) sharing updates on gene‑targeted therapies. Merit Cudkowicz, MD, MSc (Massachusetts General Hospital) highlighted biomarker‑driven trial design through MyMatch, and Avindra Nath, MD (NIH) discussed endogenous retroviruses as novel therapeutic targets. Precision medicine moved from concept to execution as leaders discussed biomarker‑driven trial design, gene‑targeted programs, and adaptive clinical strategies. Efforts such as MyMatch and Silence ALS illustrated how smarter trials can better match patients to therapies, maximizing both speed and impact.
Session VII: Clinical Trials & Expanded Access
This session focused on accelerating access and optimizing trial design. Jess Rabourn, CFA (WideTrial, Inc.) and Björn E. Oskarsson, MD (Mayo Clinic Florida) reviewed the SEANOBI‑ALS Expanded Access Program. Fred Grossman, DO (Coya Therapeutics) discussed immunomodulatory approaches, while Daniel B. Rubin, MD, PhD (Massachusetts General Hospital) explored clinical translation of brain‑computer interfaces. Merit Cudkowicz, MD, MSc provided a PREVAiLS trial update.
Session VIII: Industry Updates II
Industry partners shared late‑stage insights. Eric Green, MD, PhD (Trace Neuroscience) discussed RNA‑targeted precision medicines. Sophia Majeed, PhD (Corcept Therapeutics) provided an update on dazucorilant, and Steve Garafalo, PhD (Biogen) reflected on the development of tofersen for SOD1‑ALS and lessons for future trial design.
Special Plenary
Don Cleveland, MD (UC San Diego) delivered a landmark plenary on TDP‑43 biology, phase separation, and therapeutic targeting. This sweeping perspective on the evolution of ALS biology, highlighted how decades of foundational discovery have reshaped understanding of disease mechanisms and therapeutic opportunity. Drawing on his seminal contributions to the field, Dr. Cleveland framed key lessons from past breakthroughs while underscoring emerging directions poised to accelerate progress for people living with ALS.
Session IX: Infection, Immunity, and Inflammation
Explorations into viral triggers and immune activation challenged conventional thinking about ALS etiology. Eain Murphy, PhD (SUNY) presented evidence implicating the neurotropic virus HSV-1 in Alzheimer’s disease, highlighting how latent infections can drive long-term neuronal dysfunction. Nancy Sicotte, MD (Cedars-Sinai) reviewed data supporting Epstein–Barr virus (EBV) as a trigger for multiple sclerosis, reinforcing the concept that viral infections may initiate immune cascades with relevance to ALS. By examining infectious and inflammatory mechanisms across neurological diseases, speakers opened new avenues for therapeutic targeting and prevention strategies.
Session X: Nucleocytoplasmic Transport & TDP‑43 Pathology
The Summit concluded with a deep dive into one of ALS’s most central mechanisms. Cutting‑edge imaging, AI‑enabled analysis, and molecular genetics revealed how disruptions in nuclear transport and TDP‑43 regulation drive neuronal vulnerability—and where future therapies may intervene. Steve Finkbeiner, MD, PhD (Gladstone Institutes) demonstrated how patient-derived models, postmortem tissue, and AI uncover distinct TDP-43–associated neuronal phenotypes. Aaron D. Gitler, PhD (Stanford University) revealed previously hidden (“cryptic”) TDP-43 RNA targets, providing mechanistic insight into how transport and RNA-processing defects directly contribute to neurotoxicity. Together, these talks underscored nucleocytoplasmic transport failure as a central, actionable feature of ALS biology.
Closing
The Summit concluded with forward‑looking remarks from Clive Svendsen, PhD, Sheri Strahl, MPH, MBA, and Sarah Dougherty, PhD, reinforcing momentum toward 2027 and the continued role of ALS Network as a driver of innovation.
AWARD WINNERS:
Fred Fisher Excellence in Research Award
Dr. Jone Lopez-Erauskin from University of California, San Diego
Barber Awards
Stanislav Tsitkov from Massachusetts Institute of Technology
“A human motor-neuron QTL atlas links noncoding variation to ALS risk and progression”
He Lilian Gao from UCSD
“Mechanisms of TDP-43 Proteinopathy in Frontotemporal Dementia Determined with Multimodal Spatial Transcriptomics”
Briana Ondatje from Barrow Neurological Institute
“Molecular profiling of microglia-astrocyte crosstalk in C9orf72 ALS/FTD reveals dysregulation of critical signaling pathways’
Looking Ahead
Across three days, the Summit highlighted a field moving rapidly toward biomarker‑informed trials, AI‑enabled discovery, and precision therapeutics. ALS Network’s Research Innovation Initiative continues to play a critical role in funding and convening the science that will shape the next generation of ALS treatments. From biomarkers and AI to gene therapy, neurotechnology, and precision trials, the Summit showcased how rapidly the ALS field is evolving—and how strategic investment can accelerate real‑world impact.
The 2026 ALS Network Research Summit reaffirmed the power of convening bold thinkers across disciplines. The ideas shared—and partnerships sparked—will directly inform ALS Network’s Innovation Grant portfolio and strategic priorities, ensuring that today’s insights become tomorrow’s treatments.
Together, we are accelerating the path from discovery to impact.
