At the Vanderbilt ALS Research Center, we are dedicated to pioneering breakthroughs that advance early detection and treatment of ALS. Guided by a relentless pursuit of knowledge and grounded in compassion, we envision a future in which individuals affected by ALS are empowered by earlier interventions that meaningfully improve quality of life. Together with a community of researchers, clinicians, and advocates, we work to unravel the biological underpinnings of ALS and translate discovery into a new era of hope and impact for those living with this disease.
Research
Under the scientific leadership of Dr. Véronique Belzil, the Vanderbilt ALS Research Center brings together multiple integrated research programs focused on defining the molecular, cellular, and systems-level mechanisms underlying ALS and related neurodegenerative diseases, including frontotemporal dementia. Using deeply phenotyped human cohorts, paired ante-mortem biofluids, and post-mortem nervous system tissues, the center applies multi-omic approaches—including genomic, epigenetic, transcriptomic, and proteomic analyses—to capture disease-relevant variation across cell types, regions, and disease subgroups.
A central focus of the center’s work is understanding biological heterogeneity in ALS, including differences in site of onset, rate of progression, and underlying pathology, with the goal of identifying biologically informed disease subtypes. These efforts directly support the discovery and validation of static and dynamic biomarkers across multiple biofluids, as well as the development of clinically relevant biomarker panels to enable earlier diagnosis, improve prognostication, support patient stratification, and assess target engagement in clinical trials.
Complementing human tissue–based discovery, the center leverages human-derived cellular models to interrogate disease mechanisms, model stress and degeneration, and evaluate emerging therapeutic strategies. Through close integration of basic, translational, and clinical research programs and a highly collaborative, multidisciplinary framework, the Vanderbilt ALS Research Center is committed to accelerating the translation of discovery-driven insights into meaningful advances for individuals affected by neurodegenerative disease.
Community Outreach and Education
For information on community outreach and educational programs contact Jennifer Perfilio, Program Manager for the Vanderbilt ALS Research Center:
(615) 875-4585
For Patients
The Neuromuscular Division within the Department of Neurology at Vanderbilt University Medical Center provides comprehensive diagnosis and care for adults with nerve and muscle disorders, including amyotrophic lateral sclerosis (ALS). The multidisciplinary team works closely with individuals living with ALS to manage symptoms, optimize function, and support quality of life throughout the course of the disease.
More information on Amyotrophic Lateral Sclerosis (ALS) is available via the Vanderbilt Health Library.
Resources
