Professor of Neurology, Harvard Medical School
Director, New England Huntington's Disease Center of Excellence, Massachusetts General Hospital
Office Phone: 617-726-1254
Steven Hersch’s research focuses on Huntington’s disease (HD) and helping to develop treatments to slow or cure this fatal progressive neurogenetic disorder. His laboratory works to discover and translate potential disease modifying therapies and biomarkers from the lab to the clinic. His laboratory research has included studying the synaptic organization and molecular pharmacology of the cerebral cortex and basal ganglia; the pathophysiology of HD including mutant huntingtin protein’s toxic characteristics, oxidative stress, signal transduction, energy dysfunction, protein aggregation, proteolysis, neuroinflammation, and altered transcription; identification and validation of potential treatments for HD in mouse models; discovery, validation, and deployment of potential protein, small molecule, and transcriptomic biomarkers. The Hersch laboratory’s preclinical studies in HD mice demonstrating neuroprotective efficacy of creatine, cystamine, and phenylbutyrate have led to clinical trials in HD patients. They have tested compounds from screening efforts by colleagues at MGH and in collaborations with industry including the the first therapeutic lead for HD to move from high-throughput screening, to optimization, to proof-of-concept, to readiness for an IND. Their work on metal dysregulation in HD models and patients helped provide the rationale for a successful phase II trial of PBT2 (PRANA Biotechnology), which is now in planning for phase III. They discovered and validated H2AFY as a brain and leukocyte gene expression marker of HD progression in mice and humans and helped conceive and develop a pivotal assay for measuring the normal and mutant huntingtin proteins in models and in human blood.
Steven Hersch is a Professor of Neurology at Harvard Medical School, Attending Neurologist at Massachusetts General Hospital, Director of the New England Huntington’s disease Center of Excellence, and Director of the Laboratory of Neurodegeneration and Neurotherapeutics. He earned his Ph.D. Boston University School of Medicine (BUSM), completed a postdoctoral fellowship at Harvard Medical School, and earned his MD degree at BUMC. He completed his residency in Neurology and fellowship in Behavioral Neurology at Emory University School of Medicine where he rose to Associate Professor of Neurology. At Emory, he founded the first HDSA Huntington’s Disease Center of Excellence and led the development of this HDSA program, which now supports HD care in 26 academic clinics.