Strategies for bringing stem cell-derived dopamine neurons to the clinic:  A European approach (STEM-PD)

Agnete Kirkeby; Malin Parmar; Roger A. Barker

doi:10.1016/bs.pbr.2016.11.011

Strategies for bringing stem cell-derived dopamine neurons to the clinic: A European approach (STEM-PD)

Agnete Kirkeby, Malin Parmar, Roger A. Barker

41 Citations (Scopus)

Abstract

The treatment of Parkinson's disease (PD) has for over 50 years relied on dopaminergic therapies that are highly effective especially in the early years of the condition, but ultimately are limited by the development of side effects that relate to the nonphysiological stimulation of dopamine receptors including in nonstriatal areas. Targeted regenerative therapies designed to restore specifically the lost dopaminergic innervation of the striatum would therefore represent a major advance in treating PD. Transplantation of human fetal ventral midbrain tissue to the striatum of PD patients has provided proof-of-principle that such an approach can provide long-term clinical benefits with a reduced dependency on any oral dopaminergic agents. However, fetal tissue is associated with several ethical and logistical problems and therefore does not represent a realistic route to the clinical treatment of PD in the future. As a result, alternative cell sources have been explored and the methods for producing authentic midbrain dopaminergic neurons from pluripotent cells have now advanced to a stage which makes it possible to efficiently and reproducibly produce DA progenitors at a much higher purity than can be obtained from human fetal tissue. A stem cell-based therapy for PD therefore has the potential to circumvent many of the problems currently associated with fetal tissue grafting. Here, we describe the challenges faced and the strategies that have been pursued in our European effort to bring a human embryonic stem cell (hESC)-derived dopamine cell product to clinical trial for PD.

Original language	English
Title of host publication	Functional Neural Transplantation IV : Translation to Clinical Application, Part A
Number of pages	26
Volume	230
Publication date	2017
Pages	165-190
ISBN (Electronic)	978-0-12-811738-5
DOIs	https://doi.org/10.1016/bs.pbr.2016.11.011
Publication status	Published - 2017
Externally published	Yes

Series	Progress in Brain Research
ISSN	1875-7855

Keywords

clinical trial
dopaminergic neurons
fetal ventral mesencephalic tissue
good manufacturing practice (gmp)
human embryonic stem cells
parkinson's disease
transeuro
ventral midbrain

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access to Document

10.1016/bs.pbr.2016.11.011

Cite this

Strategies for bringing stem cell-derived dopamine neurons to the clinic : A European approach (STEM-PD). / Kirkeby, Agnete; Parmar, Malin; Barker, Roger A.

Functional Neural Transplantation IV: Translation to Clinical Application, Part A. Vol. 230 2017. p. 165-190 (Progress in Brain Research).

Research output: Chapter in Book/Report/Conference proceeding › Book chapter › Research › peer-review

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