A Report from a Workshop of the International Stem Cell Banking Initiative, Held in Collaboration of Global Alliance for iPSC Therapies and the Harvard Stem Cell Institute, Boston, 2017

Abstract

This report summarizes the recent activity of the International Stem Cell Banking Initiative held at Harvard Stem Cell Institute, Boston, MA, USA, on June 18, 2017. In this meeting, we aimed to find consensus on ongoing issues of quality control (QC), safety, and efficacy of human pluripotent stem cell banks and their derivative cell therapy products for the global harmonization. In particular, assays for the QC testing such as pluripotency assays test and general QC testing criteria were intensively discussed. Moreover, the recent activities of global stem cell banking centers and the regulatory bodies were briefly summarized to provide an overview on global developments and issues.International Stem Cell Banking Initiative (ISCBI) was established in 2007 with funding from the International Stem Cell Forum (www.iscbi.org), with the remit to support human pluripotent stem cell (PSC) banking centers, stem cell biologists, regulatory bodies, and others involved and/or interested in biobanking. The present report provides a summary of the key points of discussion from the 2017 ISCBI meeting, with emphasis on data standardization, quality control, and genetic for quality assurance and resource sharing. It provides a useful global perspective on developments in PSC applications and guidance on evaluation of emerging technologies for culture, characterization, safety testing, and ethical guidelines in the establishment of safe and effective stocks of stem cells for future regenerative medicine.

Stem‐cell–based therapies to enhance peripheral nerve regeneration

“…In addition, ethical considerations are a necessity when translating research from the bench to bedside. Furthermore, governmental restrictions and regulations may negatively affect the speed of translation of stem cell therapy into clinical practice . Strategies to manipulate cells using genetic and viral transduction approaches in vitro to potentially enhance their effect in vivo (as recently reviewed) raise additional regulatory considerations.…”

“…Furthermore, governmental restrictions and regulations may negatively affect the speed of translation of stem cell therapy into clinical practice. [156][157][158] Strategies to manipulate cells using genetic and viral transduction approaches in vitro to potentially enhance their effect in vivo (as recently reviewed 159 ) raise additional regulatory considerations. Nevertheless, the evidence presented in this review suggests an promising future for stem cell-based approaches to traumatic peripheral nerve damage, although continued high-quality research is essential for bench-tobedside translation.…”

Development of genetic quality tests for good manufacturing practice-compliant induced pluripotent stem cells and their derivatives

“…Therefore, it may be important to perform genetic quality tests on hiPSCs and differentiated cells to facilitate the selection of hiPSC seed stocks suitable for clinical application. Accordingly, there is a global consensus regarding the need to further evaluate the genomic quality testing of cell seed stocks using procedures such as karyotyping 5,7,9,10 . However, some chromosomal abnormalities are beyond the limits of resolution of routinely used assays, which may increase the risk of missing subtle chromosomal abnormalities.…”

“…However, some chromosomal abnormalities are beyond the limits of resolution of routinely used assays, which may increase the risk of missing subtle chromosomal abnormalities. Thus, it is generally agreed that more informative genomic QC tests are a prerequisite for the selection of clinical-grade hiPSCs 5,7,9,10 .There are a wide range of technological options available for monitoring the genomic integrity of clinical-grade hiPSCs, among which single-nucleotide polymorphism (SNP) chips, fluorescence in situ hybridization (FISH), whole-genome sequencing (WGS), whole-exome sequencing (WES), and karyotyping are considered appropriate methodological approaches for investigating genetic alterations in seed-stock banks 6 . In addition, gene expression profiling analysis could provide better insight into the consequences of genomic alterations 8 .…”

“…However, some chromosomal abnormalities are beyond the limits of resolution of routinely used assays, which may increase the risk of missing subtle chromosomal abnormalities. Thus, it is generally agreed that more informative genomic QC tests are a prerequisite for the selection of clinical-grade hiPSCs 5,7,9,10 .…”