title: Accelerating stem cell trials for Alzheimer's disease.
abstract: At present, no effective cure or prophylaxis exists for Alzheimer's disease. Symptomatic treatments are modestly effective and offer only temporary benefit. Advances in induced pluripotent stem cell (iPSC) technology have the potential to enable development of so-called disease-in-a-dish personalised models to study disease mechanisms and reveal new therapeutic approaches, and large panels of iPSCs enable rapid screening of potential drug candidates. Different cell types can also be produced for therapeutic use. In 2015, the US Food and Drug Administration granted investigational new drug approval for the first phase 2A clinical trial of ischaemia-tolerant mesenchymal stem cells to treat Alzheimer's disease in the USA. Similar trials are either underway or being planned in Europe and Asia. Although safety and ethical concerns remain, we call for the acceleration of human stem cell-based translational research into the causes and potential treatments of Alzheimer's disease.
authors.affiliation: Wake Forest Institute for Regenerative Medicine, Wake Forest University, Winston-Salem, NC, USA.
authors.forename: Joshua G
authors.initials: JG
authors.lastname: Hunsberger
authors.affiliation: New York Stem Cell Foundation, New York, NY, USA.
authors.forename: Mahendra
authors.initials: M
authors.lastname: Rao
authors.affiliation: Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center, Durham, NC, USA.
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.affiliation: Department of Radiology and Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
authors.forename: Jeff W M
authors.initials: JWM
authors.lastname: Bulte
authors.affiliation: Wake Forest Institute for Regenerative Medicine, Wake Forest University, Winston-Salem, NC, USA.
authors.forename: Anthony
authors.initials: A
authors.lastname: Atala
authors.affiliation: Institute for Memory Impairment and Neurological Disorders, University of California-Irvine, Irvine, CA, USA.
authors.forename: Frank M
authors.initials: FM
authors.lastname: LaFerla
authors.affiliation: Center for Law and the Biosciences, Stanford University, Stanford, CA, USA.
authors.forename: Henry T
authors.initials: HT
authors.lastname: Greely
authors.affiliation: Department of Psychiatry, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
authors.forename: Akira
authors.initials: A
authors.lastname: Sawa
authors.affiliation: Center for Cognitive Health and National Football League Neurological Care, Icahn School of Medicine at Mount Sinai, New York, NY, USA; James J Peters VA Medical Center, Bronx, NY, USA.
authors.forename: Sam
authors.initials: S
authors.lastname: Gandy
authors.affiliation: Alzheimer's Disease Research Center, Keck School of Medicine of the University of Southern California, Los Angeles, CA, USA.
authors.forename: Lon S
authors.initials: LS
authors.lastname: Schneider
authors.affiliation: Duke Institute for Brain Sciences, Duke University, Durham, NC, USA; Psychiatry Department, Duke University, Durham, NC, USA. Electronic address: murali.doraiswamy@duke.edu.
authors.forename: P Murali
authors.initials: PM
authors.lastname: Doraiswamy
mesh_terms: D000544:Alzheimer Disease / Q000628:therapy*; D002986:Clinical Trials as Topic; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000637:transplantation; D045164:Mesenchymal Stem Cell Transplantation