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

Dr. Rothstein received his BA from Colgate University. Dr. Rothstein started his graduate neuroscience training at the University of Chicago (MA, 1979) followed by his doctoral studies in biophysics and physiology and medical degree 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.

In 2000 Dr. Rothstein organized the Robert Packard Center for ALS Research at Johns Hopkins and serves as its Medical Director. This is the first multi-institutional, multi-national collaborative academic organization devoted to understanding the cause of amyotrophic lateral sclerosis 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/year. 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 other ALS and neurodegenerative disease non-profit organizations and NIH. The approach has led to the unprecedented generation of new animal models of the disease and new clinical therapeutic targets.

Dr. Rothstein's clinical specialization is in neuromuscular disease, with a particular focus on ALS. Other clinical areas relevant to his laboratory-based research include epilepsy, spinocerebellar ataxia, and brain tumors.

It was Dr. Rothstein’s research on ALS pathogenesis that led 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 to and accelerate neurodegenerative disease. His lab has spent the last 15 years using pathogenic cascade to discover new ALS therapeutics. Dr. Rothstein's Hopkins laboratory has been responsible for many ALS therapeutic candidates including small molecules, gene therapies, and more recently cellular based therapeutics.

Dr. Rothstein 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, and 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.

Dr. Rothstein's laboratory includes over 30 postdoctoral 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, the ALS Association and Project A.L.S.

Dr. Rothstein can be reached by email at jrothstein@jhmi.edu.

Dr. Rothstein's faculty home page


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