Highlights of the 14th Annual California ALS Research Summit

The 14th annual California ALS Research Summit was a seminal event in the world of ALS research and care, with over 200 researchers, investigators, clinicians, biotech companies, partner organizations, and advocates in ALS from across the state and beyond. 

The Summit serves as an annual opportunity for experts in the field to come together to discuss the latest advancements in ALS research, share their findings, and collaborate towards finding prevention strategies, effective treatments, and cures for the disease.

The two-day event was filled with presentations and discussions on a wide range of topics, from genetics to the development of new treatments. Nearly 40 guest speakers conducted special lectures, panel discussions, and scientific presentations over the two-day event. In addition to presenters from California, the Summit had participants from throughout the US.

Keynote speakers included leading ALS researchers, who shared their latest findings on the genetic basis of the disease and the role of genetics in its progression.

One of the highlights of the Summit’s first day was the presentation of alternative therapy delivery paths from Viviana Gradinaru, PhD, Director, Caltech. In recent trials, their researchers have focused on the selective permeability of the blood-brain barrier (BBB) to find barrier entry mechanisms and better understand brain diseases coupled with potential treatments. Results from non-human primate trials showed promising progress in expedited therapy delivery while addressing the variability of the BBB across different demographics.

Another exciting topic was antisense oligonucleotide (ASO) as a treatment for ALS. ASOs can target and manipulate specific parts of the cell biology and can be effective against forms of ALS where the cause has a known genetic basis. Research into ASOs is ongoing for two types of ALS, including the SOD1 and FUS mutation, an early-onset form of ALS. The discussions and presentations provided a comprehensive overview of the current state of the field and gave attendees a glimpse into the future of ALS research and treatments.

The development and progress of ASOs wouldn’t exist today without the commitment and collaboration of Richard Smith, MD, ALS Network Advisory Trustee and Director of the Center for Neurologic Study; Frank Bennett, PhD, Chief Scientific Officer at Ionis Pharmaceuticals; and Don W. Cleveland, PhD, Distinguished Professor and Chair, Department of Cellular and Molecular Medicine, UC San Diego. 

ALS Network Leadership also took a moment to acknowledge the commitment of Dr. Richard Smith, who has pledged a significant contribution of capital to help launch our new global research program. In addition to his pioneering work on antisense technology, Dr. Smith developed Nuedexta, an FDA-approved medication for ALS symptoms. He is also an ALS Network Advisory Trustee, a founding member of our Industry Partner Consortium, and a contributing member of the California ALS Research Network.

At the end of the first day, over 20 researchers participated in a robust poster presentation for the annual Barber ALS Research Awards competition. The Barber Awards were created in 2017 by the Barber family and the ALS Network in memory of Jim Barber, one of the ALS Research Summit’s founding members. It was Jim’s desire that the annual Summit would encourage collaboration and sharing of ideas as well as give special recognition to early-career researchers. Submissions are evaluated by the Chair of the ALS Network along with former Barber Award honorees. The three top poster presenters receive a special memento in recognition of their accomplishments and a cash prize of  $1000 each to support their work in ALS research.

Day two included hot topics on TDP-43, a protein that has been found in ALS patients and has links to motor neuron damage, cryptic exons, and synaptopathy in ALS.  Studying the biology of TDP-43 can provide a better understanding of sporadic ALS and what causes it.

In addition, the winners of the 7th annual Barber ALS Research Awards gave brief presentations about their work to Summit attendees. This year’s honorees include  Frederick (Eric) Arnold, PhD, of UC Irvine; Caiwei Guo, PhD, of Stanford University; and Kevin Rhine, PhD, of UCSD. Frederick elaborated on a part of the RNA process called alternative polyadenylation (APA), which is known to be regulated by TDP-43. Caiwei discussed how TDP-43 pathology triggers cryptic RNA splicing of synaptic genes in ALS/FTD. Kevin discussed how aging contributes to ALS.

Other highlights of Summit topics included 

• Important Updates from Industry and Clinical Trials
• Western ALS Consortium (WALS) Clinical Updates
• CIRM-Funded ALS Research and Clinical Trials in California
• Innovations, Modeling, and AI in ALS Research

The final session of the 14th annual Summit was entitled, “Looking Ahead: Expanding the Impact of the California ALS Research Network.” The focus of this session was on a new funding model available through the ALS Network, which could directly support new research projects. Speakers included Fred Fisher, MSW, LCSW, President & CEO, ALS Golden West; Dr. Clive Svendsen, Dr. Richard Smith, and Dr. Don Cleveland, with special remarks by Robert Bowser, PhD, CSO, Barrow Neurological Institute.

This year’s Summit was a resounding success and continues to provide a platform for the exchange of new ideas and findings among experts in the field of ALS research. With the knowledge and insights gained, we can continue to work towards finding treatments and cures for ALS and improving the lives of those affected by this devastating disease.

Highlights from the Summit are always shared with the public at our annual ASK ME webinar, “ALS Research and Care.” This year’s event, presented by Accessibility and Inclusivity sponsor Mitsubishi Tanabe Pharma America, was offered free of charge to the ALS community via Zoom and live stream. Participants have the opportunity to hear first-hand an overview of what was discussed and presented during the Summit and other important topics from top clinicians and researchers in the field.

Outcomes of the 2024 Summit were presented by Clive Svendsen, PhD, Chair, California ALS Research Network, and Jill Goslinga, MD, MPH, Assistant Professor, Neurology, UCSF Weill Institute for Neurosciences. In addition, Dr. Svendsen facilitated a discussion with the Barber ALS Research Awardees and their research on ALS and related topics. 

Our deepest gratitude to Dr. Svendsen, Dr. Goslinga, the California ALS Research Network, and all who participated in the Summit. It is only together that we will end ALS.