Accelerating stem cell trials for Alzheimer’s disease - 13/01/16

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

Joshua G Hunsberger, PhD ^a, Mahendra Rao, ProfMD ^b, Joanne Kurtzberg, ProfMD ^c, Jeff W M Bulte, ProfPhD ^d, Anthony Atala, ProfMD ^a, Frank M LaFerla, ProfPhD ^f, Henry T Greely, ProfJD ^g, Akira Sawa, ProfMD ^e, Sam Gandy, ProfMD ^h,ⁱ, Lon S Schneider, ProfMD ^j, P Murali Doraiswamy, ProfMBBS ^k,^l,^⁎

^a Wake Forest Institute for Regenerative Medicine, Wake Forest University, Winston-Salem, NC, USA

^b New York Stem Cell Foundation, New York, NY, USA

^c Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center, Durham, NC, USA

^d Department of Radiology and Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA

^e Department of Psychiatry, Johns Hopkins University School of Medicine, Baltimore, MD, USA

^f Institute for Memory Impairment and Neurological Disorders, University of California-Irvine, Irvine, CA, USA

^g Center for Law and the Biosciences, Stanford University, Stanford, CA, USA

^h Center for Cognitive Health and National Football League Neurological Care, Icahn School of Medicine at Mount Sinai, New York, NY, USA

ⁱ James J Peters VA Medical Center, Bronx, NY, USA

^j Alzheimer’s Disease Research Center, Keck School of Medicine of the University of Southern California, Los Angeles, CA, USA

^k Duke Institute for Brain Sciences, Duke University, Durham, NC, USA

^l Psychiatry Department, Duke University, Durham, NC, USA

^* Correspondence to: Prof P Murali Doraiswamy, Neurocognitive Disorders Program, Duke University Medical Center, Durham, NC 27710, USA Correspondence to: Prof P Murali Doraiswamy Neurocognitive Disorders Program Duke University Medical Center Durham NC 27710 USA

Summary

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.

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Plan

Introduction

Progress in stem cell research

Insights from modelling with iPSC-derived neurons

Specific cell types as potential therapeutics

Non-invasive methods to track stem cells and optimise cell therapy

Safety and ethical concerns

Clinical trials in neurodegenerative disorders

Outstanding questions and future perspectives

Conclusions

Search strategy and selection criteria

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Vol 15 - N° 2

P. 219-230 - février 2016 Retour au numéro

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Accelerating stem cell trials for Alzheimer’s disease - 13/01/16

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