3rd annual UC–LLNL workshop brings together researchers, clinicians, community to drive ALS innovation

In May, the third annual workshop on amyotrophic lateral sclerosis (ALS) took place at the University of California Livermore Collaboration Center (UCLCC), underscoring the growing collaboration among institutions working to address this complex neurological condition. ALS is a progressive, fatal neurodegenerative disease that attacks motor neurons, the nerve cells responsible for voluntary muscle movement and control. Although no cure exists today, work is underway to develop more effective treatments and improve patient outcomes.

The two-day event brought together approximately 90 attendees spanning the UC system's five ALS centers, DOE national laboratory researchers, industry partners, funding organizations and foundations, caregivers, and person(s) living with ALS. Co-organized by Amy Gryshuk, associate director of strategic alliances at UCSF Innovation Ventures, the third iteration of this workshop sustains the momentum in building a multidisciplinary ALS research community.

Strategic partnerships and national lab capabilities

Patricia Falcone, deputy director for science and technology at LLNL, welcomed the attendees on the first day, illuminating the role of strategic partnerships in advancing ALS research.

"Challenges like ALS require a shared effort across universities, national laboratories, industry, foundations, and the broader research ecosystem," Falcone said. "Progress depends on connecting expertise, ideas, and capabilities across institutions and sectors, because meaningful outcomes are built by teams." Falcone highlighted LLNL’s contributions across the UC system, particularly in computing, artificial intelligence, biology, bioengineering, and microfabrication. These capabilities, she noted, represent advanced physical science innovations that can be applied to pressing biological challenges as well.

Keynote speaker Richard Reimer, professor at Stanford University and chief of neurology services at the Veterans Affairs, discussed efforts to identify ALS drug repurposing candidates through analysis of electronic health records. The collaboration, led by LLNL’s Computer Engineering Division, showed how machine learning and large-scale data analysis seek to uncover off-target drug effects associated with patient survival and help guide the search for better therapies.

The first day concluded with a strategic partnerships panel featuring leaders Casper Hoogenraad from Genentech/Roche; Antoine Snijders (BBTD), division leader at LLNL; Sally Allen, of the Livermore Lab Foundation; and Mark Hettick, co-founder of Precision Neuroscience. The panelists discussed ways to catalyze multi-institution collaborations and advocated for establishing operational agreements, including data-sharing and intellectual property agreements, early in collaborative projects.

Sharing perspectives within the ALS community

Between workshop activities, participants were invited to network and explore posters highlighting the latest advances in ALS research. “We’ve focused on lightning talks and poster presentations for science and incorporating networking sessions in our workshop agenda to prompt discussion across attendees,” Gryshuk said. Nicholas Fischer (BBTD), group leader for Translational Immunology at LLNL, organized these presentations to efficiently showcase diverse research activities and expertise, followed by dedicated networking time to enable deeper discussion, connection building, and identification of synergistic opportunities. “Together, these elements promote cross-disciplinary engagement, strengthen professional networks, and facilitate the development of new partnerships and collaborative projects that can drive scientific innovation and impact,” Gryshuk said. Science topics ranged from CRISPR gene therapy, microbe characterization, brain–computer interfaces, stem-cell therapies, and efforts to improve clinical diagnosis.

A separate panel invited caregivers and people living with ALS to share first-hand perspectives on living with and caring for someone with the disease. Patients spoke candidly about their path to diagnosis, and their experiences with the disease, describing the exhaustion of fighting against overwhelming fatigue each day and the need to expedite the process for patients to see a neurologist. Their contributions underscored the need for continued research and development, as well as patient-facing tools and wearable technologies that may help shorten the path to diagnosis and improve quality of life.

Maintaining mission momentum

On the workshop’s final day, LLNL Director Kimberly Budil delivered closing remarks, emphasizing the collaborative imperative driving this work: “The era of solving major scientific problems within isolated disciplines is largely over; progress now depends on partnerships that bring together many kinds of expertise,” Budil said. “Biology, disease, and biothreats are not only scientific challenges, they are critical issues for both economic security and national security.”

The workshop concluded with a funding panel, “Driving Discovery: Navigating ALS Funding,” featuring representatives from organizations such as Target ALS, the National Institute of Neurological Disorders and Stroke at the National Institutes of Health, ALS Network, the California Institute for Regenerative Medicine, and Breakout Ventures. The session created space for attendees to explore potential partnerships and learn about funding mechanisms across government, nonprofit, and private investment sectors.

Looking ahead, workshop organizers hope to broaden participation by engaging additional University of California campuses, colleagues across the DOE complex, and more industry and investment partners. They also plan to place greater emphasis on involving and uplifting early-career researchers to help strengthen and sustain momentum in the field.

—Grace Jeng