Dr. Jeffrey D. Rothstein stands as a preeminent neuroscientist whose pioneering work has significantly advanced our understanding of amyotrophic lateral sclerosis and related neurodegenerative disorders. He currently serves as the John W. Griffin Director of the Brain Science Institute and Professor of Neurology and Neuroscience at Johns Hopkins University School of Medicine, where he has established himself as a transformative leader in neurological research. Dr. Rothstein founded and directs the Robert Packard Center for ALS Research, the first multi-institutional, multi-national collaborative academic organization dedicated to understanding ALS causes and developing innovative therapies. In 2000, he organized this groundbreaking center, creating a new paradigm for collaborative research in neurodegenerative diseases. Additionally, he maintains an active clinical practice at Johns Hopkins, evaluating and managing care for over 350 ALS patients annually, which informs his translational research approach.
Dr. Rothstein's seminal research on ALS pathogenesis directly contributed to the development of the first FDA-approved drug capable of altering neurodegeneration in ALS patients, representing a watershed moment in the field. His laboratory has made fundamental contributions to understanding astrocyte and oligodendroglial biology in both healthy and diseased states, particularly focusing on glutamate transporter mechanisms that are critical for neuronal survival. He has pioneered research into nuclear pore biology, nucleoporins, and nuclear transport defects in ALS, Huntington's disease, and dementias, opening new avenues for therapeutic intervention. Dr. Rothstein's innovative work with human induced pluripotent stem cells has enabled unprecedented study of ALS and frontotemporal dementia pathophysiology in human-derived cellular models. His research program employs comprehensive multi-omics analytics including whole genome, epigenome, RNA profiling, and proteome studies to identify novel therapeutic targets for neurodegenerative conditions.
As Founder and Executive Director of the Answer ALS Research Program, Dr. Rothstein has spearheaded one of the most comprehensive ALS research initiatives, generating over 1,000 ALS and control induced pluripotent stem cell lines that are extensively characterized through multi-omics approaches. He directs the Brain Science Institute, which serves as a critical nexus for bridging basic and clinical neuroscience and houses a robust drug discovery group focused on identifying novel therapeutics for neurological and psychiatric disorders. His laboratory utilizes both high and low throughput screening systems with chemical libraries to identify compounds that activate EAAT genes for potential therapeutic applications in ALS. Current research directions include neuroprotective studies using viral vector delivery approaches, neural and astroglial stem cell therapies, and exploring glial progenitors as neuroprotectants in ALS-relevant models. Through his visionary leadership and translational research paradigm, Dr. Rothstein continues to drive innovation in understanding and treating neurodegenerative diseases, positioning his work at the forefront of bringing laboratory discoveries to patient care.
