Developing safe therapies from human pluripotent stem cells

Carpenter, Melissa K.; Frey-Vasconcells, Joyce; Rao, Mahendra S.

doi:10.1038/nbt0709-606

Cited by 128 publications

(84 citation statements)

References 76 publications

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“…Many common issues dealing with cell transplantation have been addressed (for more detail see Carpenter et al [11] and Ahrlund-Richter et al [12]). We will focus on issues that are relevant to transplanting cells into the CNS.…”

Section: Issues Of High Importance To the Treatment Of Neural Disordersmentioning

confidence: 99%

Cell-Based Therapy for Neural Disorders — Anticipating Challenges

Chiu

Rao

2011

Neurotherapeutics

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Neurological syndromes, such as Alzheimer's disease, Parkinson's disease, multiple sclerosis, Huntington's disease, amyotrophic lateral sclerosis, and lysosomal storage disorders, such as Battens disease, are devastating because they result in increasing loss of cognitive and physical function. Sadly, no drugs are currently available to halt their progression. The relative paucity of curative approaches for these and other conditions of the nervous system have led to a widespread evaluation of alternative treatment modalities including cell-based interventions.Several cell types have been tested successfully in animal models where safety and efficacy have been demonstrated. Early clinical trials have also been initiated in humans, and some have shown a degree of success albeit on a more limited scale than in animal experiments. Recent demonstrations that pluripotent stem cells, such as embryonic stem cells and induced pluripotent stem cells, can differentiate into a variety of specific neural phenotypes has stimulated worldwide enthusiasm for developing cell-based intervention of neurological disease. Indeed, several groups are preparing investigational new drug applications to treat disorders as diverse as macular degeneration, lysosomal storage diseases, and Parkinson's disease. It is noteworthy that cell replacement therapies for neurological conditions face key challenges, some of which are unique, because of the development and organization of the nervous system, its metabolism, and connectivity. Choice of the cell (or cells), the process of manufacturing them, defining the delivery pathway, developing and testing in an appropriate preclinical model, selecting a patient population, and visualizing and following or monitoring patients all pose specific issues as related to the central and peripheral nervous systems. In this review, we address a myriad of challenges that are solvable, but require careful planning and attention to the special demands of the human nervous system.

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Section: Issues Of High Importance To the Treatment Of Neural Disordersmentioning

confidence: 99%

Cell-Based Therapy for Neural Disorders — Anticipating Challenges

Chiu

Rao

2011

Neurotherapeutics

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“…These valuable features make them ideal starting materials for developing scalable commercial cell products (17). However, manufacturing clinical-grade stem cell products for a clinical trial presents a number of difficult challenges that are not present in a research setting.…”

Section: Tumorigenicity Studiesmentioning

confidence: 99%

Hurdles to clinical translation of human induced pluripotent stem cells

Neofytou¹,

O’Brien²,

Couture³

et al. 2015

J. Clin. Invest.

139

138

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“…The safety studies of PSC-derived products include the evaluation of toxicity, tumorigenicity, and bio-distribution. The guidelines of cGMP as described in Title 21 CFR 210, 211, 312, and 600 provide regulations for screening, testing, processing, labeling, and packaging of the products (Carpenter et al 2009). …”

Section: Regulations and Guidelines For Stem Cell-derived Productsmentioning

confidence: 99%

Stem cell bioprocess engineering towards cGMP production and clinical applications

Sart

Schneider

et al. 2014

Cytotechnology

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Stem cells, including mesenchymal stem cells and pluripotent stem cells, are becoming an indispensable tool for various biomedical applications including drug discovery, disease modeling, and tissue engineering. Bioprocess engineering, targeting large scale production, provides a platform to generate a controlled microenvironment that could potentially recreate the stem cell niche to promote stem cell proliferation or lineage-specific differentiation. This survey aims at defining the characteristics of stem cell populations currently in use and the present-day limits in their applications for therapeutic purposes. Furthermore, a bioprocess engineering strategy based on bioreactors and 3-D cultures is discussed in order to achieve the improved stem cell yield, function, and safety required for production under current good manufacturing practices.

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Developing safe therapies from human pluripotent stem cells

Cited by 128 publications

References 76 publications

Cell-Based Therapy for Neural Disorders — Anticipating Challenges

Cell-Based Therapy for Neural Disorders — Anticipating Challenges

Hurdles to clinical translation of human induced pluripotent stem cells

Stem cell bioprocess engineering towards cGMP production and clinical applications

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