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University of Pittsburgh

Directors

Center Co-Director

Robert Ferrante, PhD, MS

Robert Ferrante, PhD, MS

Dr. Robert Ferrante, Co-Director, is a Visiting Professor of Neurological Surgery, Neurology, and Neurobiology at the University of Pittsburgh School of Medicine. He is the director of the Experimental Neuropathology and Translational Therapeutics Laboratory in the Neurological Surgery Department. Prior to coming to Pittsburgh in 2011, Dr. Ferrante spent over 20 years at Harvard Medical School and the Massachusetts General Hospital in Boston. His work there brought to light and strengthened current hypotheses of cell death. In 1993 he accepted a position at the Boston University School of Medicine and the Boston Veterans Administration System where he developed a bench-to-bedside program to treat neurological conditions. Dr. Ferrante has a wide-range of knowledge about the neuropathology and mechanisms of neurodegeneration in adult-onset neurological diseases, especially ALS. He is considered an expert in the application of experimental models of disease and in bench to bedside translational studies. As an international scholar in ALS, Dr. Ferrante collaborates with other researchers and clinicians worldwide in trying to find the cause and treatment for this dreaded disease. He has been a Director and Principal Proponent in human clinical trials for ALS patients, using a novel drug agent established in his laboratory. Dr. Ferrante is a founding member and remains on the Board of Directors of the ALS Therapy Alliance http://www.alstherapyalliance.org/about/directors-staff.html andis a longstanding member of the Northeast ALS Consortium http://www.alsconsortium.org/. He is a steering committee member on six current human clinical trials using therapeutic agents that were developed in his laboratories. He also holds a position in the Veterans Administration Pittsburgh Healthcare System, where he is involved in ALS research in understanding the causes of the disease. Over the past 20 years, Dr. Ferrante has developed one of the premier translational programs for understanding pathophysiological mechanisms of disease and for developing and characterizing therapeutic strategies for neurological diseases. His work has set the standard for others in the field. His laboratory has been a driving force in completing pre-clinical drug trials in mice for direct translation to human clinical trials in ALS patients. His discoveries have led to human clinical trials using coenzyme Q10, creatine, sodium phenylbutyrate, and minocycline. A high dose creatine trial in ALS patients is now under way in collaboration with other investigators at Harvard University Medical School. Dr. Ferrante currently has two funded grants investigating central and peripheral biomarkers of ALS and a bench to bedside program in drug development for ALS. The goal is to find a unique, early diagnostic biochemical signature relating to ALS subjects and to evaluate correlative biomarkers and biomarkers in response to therapy that would provide a powerful means to assess therapeutic treatments in ALS subjects and to predict the potential magnitude of benefits. In addition, Dr. Ferrante has an ongoing drug discovery program developing novel drug agents that are directed at a number of the potential causes of ALS. This work has recently led to a compound that results in a 30 percent increase in the life span of experimental ALS mice. If these were to be equated with human ALS, the effective administration of this drug may potentially add a significant number of years of life to patients. This work is to be highlighted by the Department of Defense. Along with Dr. David Lacomis, the medical director of the Center for ALS Research, the intent is to develop a research and clinical program second to none at the University of Pittsburgh, providing the best possible care for ALS patients.

Center Co-Director

David Lacomis, MD

David Lacomis, MD

Dr. Lacomis is a Professor of Neurology and Pathology as well as Chief, Division of Neuromuscular Diseases. He directs the MDA-ALS Center and multidisciplinary clinic, co-directs the electromyography laboratory, and is in charge of the muscle and nerve pathology service. Dr. Lacomis has been named annually among the “Top Doctors” by Pittsburgh magazine, and in both “Best Doctors” and “America’s Top Doctors” nationally. He lectures extensively and is a contributing author for numerous medical journals and books regarding his major clinical interests of ALS, myasthenia gravis, and muscle diseases. Dr. Lacomis is the site Principal Investigator (PI) in the following studies: (1) a Phase III study of dexpramipexole (R(+) pramipexole) for ALS (2) a multicenter study for the validation of ALS biomarkers and (3) a phase III study of ceftriaxone in ALS (4) the continuation of a phase II trial of KNS-760704-CL201 (dexpramipexole) that is now in an open-label phase, and he will also be the site PI for a second phase III study of dexpramipexole. He is also an investigator in a study assessing use of BiPAP and an epidemiology study in ALS. He is also a participant in the PA ALS Registry. With Dr. Robert Bowser, he has extensively studied biomarkers in the cerebrospinal fluid and serum in ALS patients compared to controls, trying to find a diagnostic panel and to identify disease-related changes. He also serves as the Director of the Clinical Neurophysiology fellowship, teaching and mentoring clinical fellows, medical students and house staff from Neurology and Pathology. He serves on the Clinical Neurophysiology Examination Committee for the American Board of Psychiatry and Neurology. Dr. Lacomis is a member of the editorial board for the Journal of Clinical Neuromuscular Diseases and two scientific advisory committees. He is dedicated to expanding translational and clinical research in ALS in Pittsburgh to a leadership level in collaboration with Dr. Ferrante. See Publications

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