Concise Review: Making and Using Clinically Compliant Pluripotent Stem Cell Lines

Abstract

The field of pluripotent stem cells (PSCs) is in a state of dynamic flux driven by significant advances in the derivation of specific phenotypes from embryonic stem cells, breakthroughs in somatic cell nuclear transfer, and dramatic improvements in generating induced PSCs using zero footprint methods. Spurred by these technological advances, companies have begun to plan clinical studies using human PSC derivatives manufactured in current Good Manufacturing Practice-compliant conditions. In the present review, we discuss the challenges in making these biological products, starting from tissue sourcing to the processes involved in manufacture, storage, and distribution. Additional challenges exist to meeting the regulatory requirements and keeping costs affordable. A model is described that has been proposed by the U.S. National Institutes of Health for reducing the costs and permitting flexibility and innovation by individual investigators. This model, combined with small adjustments in the regulatory processes tailored to address the unique properties of PSCs, has the potential of significantly accelerating the implementation of PSC-based cell therapy. STEM CELLS TRANSLATIONAL MEDICINE 2015;4:381-388

PAX7 Targets, CD54, Integrin α9β1, and SDC2, Allow Isolation of Human ESC/iPSC-Derived Myogenic Progenitors

“…This has the potential to revolutionize the field of regenerative medicine as it potentially enables for the use of autologous genome-edited or allogeneic HLA-matched iPS cells. Importantly, the future success of regenerative medicine relies on the safe and reproducible production of tissue-specific progenitors from PS cells (Carpenter and Rao, 2015). Methods for the isolation of the target cell population represent one of the crucial requirements for clinical application of PS cell-derived stem cell products, and their development requires a detailed characterization of the PS cell differentiation process.…”

Pluripotent Stem Cell-Derived Pancreatic Progenitors and β-Like Cells for Type 1 Diabetes Treatment

“…The length and efficiency of differentiation is key for the development of cell therapy products, which requires manufacturing, storage, and distribution in accordance with GMP compliant practices (13,71). In vitro differentiation of hPSCs to PPs takes~2 wk and generates relatively homogenous populations of PPs (43,45,57,66) or a combination of PPs and CHGAϩ cells, rendering this approach scalable and cost effective (32).…”

Developing Induced Pluripotent Stem Cell-Based Therapy for the Masses

“…However, Europe has developed a new Advanced Therapy Medicinal Product classification system and a unified submission process [11], which likely will enable costs reductions. In other countries, such as Japan, additional costs reductions and acceleration can be obtained because of the legislative changes that have been implemented [12]. Perhaps GAIT [13], a recently formed nonprofit organization to help develop IPSC‐based therapy as an international harmonized effort, offers the best hope of making iPSC‐based therapy globally viable.…”