title: From Dish to Bedside: Lessons Learned While Translating Findings from a Stem Cell Model of Disease to a Clinical Trial.
abstract: While iPSCs have created unprecedented opportunities for drug discovery, there remains uncertainty concerning the path to the clinic for candidate therapeutics discovered with their use. Here we share lessons that we learned, and believe are generalizable to similar efforts, while taking a discovery made using iPSCs into a clinical trial.
authors.affiliation: Regenerative Medicine Discovery Performance Unit, GlaxoSmithKline, Cambridge, MA 02139, USA. Electronic address: john.d.mcneish@gsk.com.
authors.forename: John
authors.initials: J
authors.lastname: McNeish
authors.affiliation: Regenerative Medicine Discovery Performance Unit, GlaxoSmithKline, Cambridge, MA 02139, USA.
authors.forename: Jason P
authors.initials: JP
authors.lastname: Gardner
authors.affiliation: Departments of Neurology and Anesthesia, Critical Care and Pain Medicine, Massachusetts General Hospital, Boston, MA 02114, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
authors.forename: Brian J
authors.initials: BJ
authors.lastname: Wainger
authors.affiliation: Harvard Stem Cell Institute, Cambridge, MA 02138, USA; FM Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
authors.forename: Clifford J
authors.initials: CJ
authors.lastname: Woolf
authors.affiliation: Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Howard Hughes Medical Institute and Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02139, USA. Electronic address: eggan@mcb.harvard.edu.
authors.forename: Kevin
authors.initials: K
authors.lastname: Eggan
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy / Q000235:genetics / Q000503:physiopathology; D000818:Animals; D002219:Carbamates / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D055808:Drug Discovery; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000187:drug effects / Q000502:physiology; D051379:Mice; D008959:Models, Neurological; D009046:Motor Neurons / Q000187:drug effects / Q000502:physiology; D009154:Mutation; D010655:Phenylenediamines / Q000627:therapeutic use; D013482:Superoxide Dismutase / Q000235:genetics; D000072105:Superoxide Dismutase-1; D057170:Translational Research, Biomedical
chemical_list: D002219:Carbamates; D010655:Phenylenediamines; C000606290:SOD1 protein, human; C101866:ezogabine; C000606291:Sod1 protein, mouse; D013482:Superoxide Dismutase; D000072105:Superoxide Dismutase-1