Jeffrey D. Rothstein, M.D., Ph.D.

Dr. Rothstein is Professor of Neurology and Neuroscience and a faculty member of the Graduate Program in Cellular and Molecular Medicine at Johns Hopkins University.  He is the Director of the Robert Packard Center for ALS Research at Johns Hopkins, and he is the Co-Director of the MDA/ALS Clinic, and Vice Chairman for Research in the Department of Neurology. He oversees one of the largest ALS clinics in the USA.

He received his BA from Colgate University.  He started his graduate neuroscience training at the University of Chicago (MA, 1979) followed by his Doctoral studies in biophysics and physiology and Medical degrees from the University of Illinois (1984, 1985). Following an Internship in medicine at the University of North Carolina, Chapel Hill, he moved to Johns Hopkins in 1986, for a Neurology Residency. Following his Neurology training, and fellowship in Neuromuscular disease, he stayed on as faculty. He currently is Professor of Neurology and Neuroscience and the Program in Cellular and Molecular Medicine. 

In 2000 Dr. Rothstein organized the Robert Packard Center for ALS Research at Johns Hopkins and serves as Medical Director.  This is the first multi-institutional, multi-national collaborative academic organization devoted toward understanding the cause of ALS and translating the information into new drug and cell based therapies. It uses an aggressive model of funding research among the leading young and senior researchers with funding based on performance expectations and mandatory collaboration.  Currently the Center funds approximately 30 researchers, spending $2-3 million/yr. In the last 5 years the vast majority of leading ALS achievements, by researchers from around the country, has been the result of the various investigators supported via this approach. In recent years the collaboration has been extended to ALS and Neurodegenerative Disease non-profit organizations and NIH. The approach has lead to the unprecedented generation of new animal models of the disease and new clinical therapeutic targets.

His clinical specialization is in Neuromuscular disease, with a particular focus on amyotrophic lateral sclerosis. Other clinical areas relevant to his laboratory-based research include: epilepsy and spinocerebellar ataxia, and brain tumors.

His basic laboratory research includes: 1) regulation of glutamate transporter by protein-protein interactions and genomic processing, 2) role of transporter regulation in abnormal synaptic transmission and neurodegeneration, 3) the biology of astroglial in neurodegenerative disease, 4) various molecular mechanisms of selective neurodegeneration in motor neuron disease, 5) identification drugs and non-chemical means to modulate glutamate transporters as therapeutics, 6) the cloning and characterization of novel proteins which may be responsible for the cellular regulation of all glutamate transporter subtypes; 7) the role of astrocytes and memebrane transporters in normal and abnormal synaptic transmission.

His pre-clinical research program includes: 1) identification of novel drug or peptide therapeutics to delay or prevent motor neuron degeneration in ALS thru the use of cell culture and transgenic models of ALS; 2) use of neuronal and non-neuronal stem cell therapies to treat motor neuron diseases including ALS; 3) models of motor axon regeneration/regrowth; 5) Clinical trials of novel therapeutics in ALS. 

It was his research on ALS pathogenesis that lead to the first successful, FDA approved drug to alter neurodegeneration in ALS. His work on glutamate transporters and astroglial dysfunction was the first evidence that glial dysfunction could contribute and accelerate neurodegenerative disease.  His lab has spent the last 15 years using pathogenic cascade to discover new ALS therapeutics.  The Hopkins laboratory has been responsible for many ALS therapeutic candidates including small molecules, gene therapies, and more recently cellular based therapeutics.

His work of basic synaptic proteins, glutamate transporter has lead to a critical understanding on the normal regulation of the excitatory neurotransmitter glutamate- but also how these classes of protein can contribute to various acute and chronic neurological insults including the growth and spread of brain tumors, neurodegeneration associated with ALS, Alzheimer’s disease and spinocerebellar ataxia. Recently, thru studies in his lab, proteins that regulate glutamate transport were found to be the cause of  Spinocerebellar ataxia 5- the disease that affected Abraham Lincoln’s family.

He has received numerous awards for his work on ALS, including the Sheila Essay award-recognizing his worldwide contribution to ALS research, as well as the Diamond Award for ALS research, the Lois Pope Foundation award for medical research, The Landa Foundation Lectureship.

He has received and/or submitted almost a dozen patents applications based on his laboratory research.  He is the Co-Founder of Ruxton Pharmaceuticals, Inc and serves as its scientific advisor.

His laboratory includes over 30 Post-doctoral fellows, neurology residents, Neuromuscular fellows, undergraduate students, technicians and ALS clinic staff. He is the Co-Director of the MDA/ALS Clinic and has been the Principal and/or Local investigator in almost a dozen National or International trials in ALS. He is the author of over 150 articles on basic and clinical neuroscience.

Dr. Rothstein’s laboratory research is funded through the National Institutes of Health, the Muscular Dystrophy Association, ALS Association and  Project A.L.S.

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