title: Clinical Trials in a Dish: The Potential of Pluripotent Stem Cells to Develop Therapies for Neurodegenerative Diseases.
abstract: Neurodegenerative diseases are a leading cause of death. No disease-modifying therapies are available, and preclinical animal model data have routinely failed to translate into success for therapeutics. Induced pluripotent stem cell (iPSC) biology holds great promise for human in vitro disease modeling because these cells can give rise to any cell in the human brain and display phenotypes specific to neurodegenerative diseases previously identified in postmortem and clinical samples. Here, we explore the potential and caveats of iPSC technology as a platform for drug development and screening, and the future potential to use large cohorts of disease-bearing iPSCs to perform clinical trials in a dish.
authors.affiliation: Gladstone Institute of Neurological Disease, San Francisco, California 94158; email: sfinkbeiner@gladstone.ucsf.edu.
authors.forename: Kelly M
authors.initials: KM
authors.lastname: Haston
authors.affiliation: Gladstone Institute of Neurological Disease, San Francisco, California 94158; email: sfinkbeiner@gladstone.ucsf.edu.|Taube/Koret Center for Neurodegenerative Disease and the Hellman Family Foundation Program in Alzheimer's Disease Research, San Francisco, California 94158.|Departments of Neurology and Physiology, University of California, San Francisco, California 94143.
authors.forename: Steven
authors.initials: S
authors.lastname: Finkbeiner
mesh_terms: D000818:Animals; D001921:Brain / Q000503:physiopathology; D002986:Clinical Trials as Topic; D006801:Humans; D019636:Neurodegenerative Diseases / Q000503:physiopathology*; D039904:Pluripotent Stem Cells / Q000502:physiology*
keywords: clinical trials; drug development; human pluripotent stem cells; neurodegeneration