Accelerating stem cell trials for Alzheimer's disease

Joshua G Hunsberger; Mahendra Rao; Joanne Kurtzberg; Jeff W M Bulte; Anthony Atala; Frank M LaFerla; Henry T Greely; Akira Sawa; Sam Gandy; Lon S Schneider; P Murali Doraiswamy

doi:10.1016/S1474-4422(15)00332-4

Accelerating stem cell trials for Alzheimer's disease

Lancet Neurol. 2016 Feb;15(2):219-230. doi: 10.1016/S1474-4422(15)00332-4. Epub 2015 Dec 17.

Authors

Affiliations

¹ Wake Forest Institute for Regenerative Medicine, Wake Forest University, Winston-Salem, NC, USA.
² New York Stem Cell Foundation, New York, NY, USA.
³ Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center, Durham, NC, USA.
⁴ Department of Radiology and Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
⁵ Institute for Memory Impairment and Neurological Disorders, University of California-Irvine, Irvine, CA, USA.
⁶ Center for Law and the Biosciences, Stanford University, Stanford, CA, USA.
⁷ Department of Psychiatry, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
⁸ 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.
⁹ Alzheimer's Disease Research Center, Keck School of Medicine of the University of Southern California, Los Angeles, CA, USA.
¹⁰ Duke Institute for Brain Sciences, Duke University, Durham, NC, USA; Psychiatry Department, Duke University, Durham, NC, USA. Electronic address: murali.doraiswamy@duke.edu.

PMID: 26704439
DOI: 10.1016/S1474-4422(15)00332-4

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.

Publication types

Review
Research Support, U.S. Gov't, Non-P.H.S.
Research Support, Non-U.S. Gov't
Research Support, N.I.H., Extramural

MeSH terms

Alzheimer Disease / therapy*
Clinical Trials as Topic*
Humans
Induced Pluripotent Stem Cells* / transplantation
Mesenchymal Stem Cell Transplantation*

Abstract

Publication types

MeSH terms

Grants and funding