Points to consider in the development of seed stocks of pluripotent stem cells for clinical applications: International Stem Cell Banking Initiative (ISCBI).

In 2009 the International Stem Cell Banking Initiative (ISCBI) contributors and the Ethics Working Party of the International Stem Cell Forum published a consensus on principles of best practice for the procurement, cell banking, testing and distribution of human embryonic stem cell (hESC) lines for research purposes [1], which was broadly also applicable to human induced pluripotent stem cell (hiPSC) lines. Here, we revisit this guidance to consider what the requirements would be for delivery of the early seed stocks of stem cell lines intended for clinical applications. The term ‘seed stock’ is used here to describe those cryopreserved stocks of cells established early in the passage history of a pluripotent stem cell line in the lab that derived the line or a stem cell bank, hereafter called the ‘repository’.

[1]  H. Shaughnessy Rules and guidance for pharmaceutical manufacturers and distributors , 2016 .

[2]  N. Benvenisty,et al.  Aneuploidy induces profound changes in gene expression, proliferation and tumorigenicity of human pluripotent stem cells , 2014, Nature Communications.

[3]  S. Yamanaka,et al.  Harmonizing standards for producing clinical-grade therapies from pluripotent stem cells , 2014, Nature Biotechnology.

[4]  A. Hoffman,et al.  Rapid and Efficient Conversion of Integration-Free Human Induced Pluripotent Stem Cells to GMP-Grade Culture Conditions , 2014, PloS one.

[5]  Sara Chandros Hull,et al.  Identifiability and privacy in pluripotent stem cell research. , 2014, Cell stem cell.

[6]  Sicong Zeng,et al.  Identification of Proteins Related to Epigenetic Regulation in the Malignant Transformation of Aberrant Karyotypic Human Embryonic Stem Cells by Quantitative Proteomics , 2014, PloS one.

[7]  M. Mandai,et al.  Tumorigenicity Studies of Induced Pluripotent Stem Cell (iPSC)-Derived Retinal Pigment Epithelium (RPE) for the Treatment of Age-Related Macular Degeneration , 2014, PloS one.

[8]  R. McKay,et al.  Human pluripotent stem cell culture: considerations for maintenance, expansion, and therapeutics. , 2014, Cell stem cell.

[9]  Y. Hoki,et al.  Induced Pluripotent Stem Cell Generation-Associated Point Mutations Arise during the Initial Stages of the Conversion of These Cells , 2014, Stem cell reports.

[10]  M. Riegel Human molecular cytogenetics: From cells to nucleotides , 2013, Genetics and molecular biology.

[11]  J. Loring,et al.  Assessment of human pluripotent stem cells with PluriTest , 2014 .

[12]  Amit C. Nathwani,et al.  A Systematic Evaluation of Integration Free Reprogramming Methods for Deriving Clinically Relevant Patient Specific Induced Pluripotent Stem (iPS) Cells , 2013, PloS one.

[13]  M. Nykter,et al.  Karyotypically abnormal human ESCs are sensitive to HDAC inhibitors and show altered regulation of genes linked to cancers and neurological diseases. , 2013, Stem cell research.

[14]  R. Lothe,et al.  BCL-XL Mediates the Strong Selective Advantage of a 20q11.21 Amplification Commonly Found in Human Embryonic Stem Cell Cultures , 2013, Stem cell reports.

[15]  M. Rao,et al.  The DISCUSS Project: Induced Pluripotent Stem Cell Lines From Previously Collected Research Biospecimens and Informed Consent: Points to Consider , 2013, Stem cells translational medicine.

[16]  P. Tan,et al.  Molecular cytogenetics: recent developments and applications in cancer , 2013, Clinical genetics.

[17]  Yi Zhang,et al.  Genetic and epigenetic variations in iPSCs: potential causes and implications for application. , 2013, Cell stem cell.

[18]  F. Müller,et al.  Reconsidering pluripotency tests: do we still need teratoma assays? , 2013, Stem cell research.

[19]  D. Bonatto,et al.  Reviewing and updating the major molecular markers for stem cells. , 2013, Stem cells and development.

[20]  E. Benson,et al.  Translating cryobiology principles into trans-disciplinary storage guidelines for biorepositories and biobanks: a concept paper. , 2013, Cryo letters.

[21]  Yoav Mayshar,et al.  Virtual karyotyping of pluripotent stem cells on the basis of their global gene expression profiles , 2013, Nature Protocols.

[22]  G. J. Morris,et al.  Controlled ice nucleation in cryopreservation--a review. , 2013, Cryobiology.

[23]  Brigitte Schlegelberger,et al.  Genetic instability of modified stem cells - a first step towards malignant transformation? , 2013, American journal of stem cells.

[24]  C. Spits,et al.  Genetic and epigenetic instability in human pluripotent stem cells. , 2013, Human reproduction update.

[25]  Karen S. Frese,et al.  Next-Generation Sequencing: From Understanding Biology to Personalized Medicine , 2013, Biology.

[26]  Simona Soverini,et al.  Comparison of Next-Generation Sequencing Systems , 2013 .

[27]  Eran Halperin,et al.  Identifying Personal Genomes by Surname Inference , 2013, Science.

[28]  Yi Zhang,et al.  Embryonic stem cell and induced pluripotent stem cell: an epigenetic perspective , 2012, Cell Research.

[29]  Benjamin E. Reubinoff,et al.  Standardization of the Teratoma Assay for Analysis of Pluripotency of Human ES Cells and Biosafety of Their Differentiated Progeny , 2012, PloS one.

[30]  P. Andrews,et al.  Deficient DNA Damage Response and Cell Cycle Checkpoints Lead to Accumulation of Point Mutations in Human Embryonic Stem Cells , 2012, Stem cells.

[31]  Vincent C. Chen,et al.  Epigenetic stability, adaptability, and reversibility in human embryonic stem cells , 2012, Proceedings of the National Academy of Sciences.

[32]  Javier Martin,et al.  Investigation of porcine circovirus contamination in human vaccines. , 2012, Biologicals (Print).

[33]  Wenxiu Zhao,et al.  Embryonic Stem Cell Markers , 2012, Molecules.

[34]  B. Knoppers,et al.  Disclosure and management of research findings in stem cell research and banking: policy statement. , 2012, Regenerative medicine.

[35]  Julie V. Harness,et al.  Recurrent variations in DNA methylation in human pluripotent stem cells and their differentiated derivatives. , 2012, Cell stem cell.

[36]  Yang Xu,et al.  The genomic stability of induced pluripotent stem cells , 2012, Protein & Cell.

[37]  R. Rao,et al.  Analysis of Embryoid Bodies Derived from Human Induced Pluripotent Stem Cells as a Means to Assess Pluripotency , 2012, Stem cells international.

[38]  F. Pistollato,et al.  Standardization of pluripotent stem cell cultures for toxicity testing , 2012, Expert opinion on drug metabolism & toxicology.

[39]  R. Xu,et al.  Concise Review: Genomic Stability of Human Induced Pluripotent Stem Cells , 2012, Stem cells.

[40]  Dong Ryul Lee,et al.  Screening ethnically diverse human embryonic stem cells identifies a chromosome 20 minimal amplicon conferring growth advantage , 2011, Nature Biotechnology.

[41]  L. Laurent,et al.  Normal Human Pluripotent Stem Cell Lines Exhibit Pervasive Mosaic Aneuploidy , 2011, PloS one.

[42]  A. Courtney,et al.  The Procurement of Cells for the Derivation of Human Embryonic Stem Cell Lines for Therapeutic Use: Recommendations for Good Practice , 2011, Stem Cell Reviews and Reports.

[43]  B. Oliver,et al.  Microarrays, deep sequencing and the true measure of the transcriptome , 2011, BMC Biology.

[44]  S. Yamanaka,et al.  The Use of Induced Pluripotent Stem Cells in Drug Development , 2011, Clinical pharmacology and therapeutics.

[45]  Jeanne F. Loring,et al.  A call for standardized naming and reporting of human ESC and iPSC lines. , 2011, Cell stem cell.

[46]  K. Brorson,et al.  Mycoplasma testing of cell substrates and biologics: Review of alternative non-microbiological techniques. , 2011, Molecular and cellular probes.

[47]  Charles J. Hunt,et al.  Cryopreservation of Human Stem Cells for Clinical Application: A Review , 2011, Transfusion Medicine and Hemotherapy.

[48]  R. Nims,et al.  Authentication of human cell-based products: the role of a new consensus standard. , 2011, Regenerative medicine.

[49]  Bernhard M. Schuldt,et al.  A bioinformatic assay for pluripotency in human cells , 2011, Nature Methods.

[50]  Michael J. Ziller,et al.  Reference Maps of Human ES and iPS Cell Variation Enable High-Throughput Characterization of Pluripotent Cell Lines , 2011, Cell.

[51]  D. Lawrence Stem Cells in Toxicology , 2011, Current protocols in toxicology.

[52]  E. Kirkness,et al.  Somatic coding mutations in human induced pluripotent stem cells , 2011, Nature.

[53]  Julie V. Harness,et al.  Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture. , 2011, Cell stem cell.

[54]  P. Stanier,et al.  The effects of culture on genomic imprinting profiles in human embryonic and fetal mesenchymal stem cells , 2011, Epigenetics.

[55]  Charles M Strom,et al.  Spectral Karyotyping for identification of constitutional chromosomal abnormalities at a national reference laboratory , 2012, Molecular Cytogenetics.

[56]  B. Knoppers,et al.  Publishing SNP Genotypes of Human Embryonic Stem Cell Lines: Policy Statement of the International Stem Cell Forum Ethics Working Party , 2011, Stem Cell Reviews and Reports.

[57]  E. Volpi,et al.  An Improved Technique for Chromosomal Analysis of Human ES and iPS Cells , 2010, Stem Cell Reviews and Reports.

[58]  D. Collins,et al.  143. Optimisation of current Good Manufacturing Practice (cGMP) compliant controlled rate freezing for human embryonic stem cells , 2010 .

[59]  G. Lin,et al.  Differences between karyotypically normal and abnormal human embryonic stem cells , 2010, Cell proliferation.

[60]  F. Müller,et al.  A call to standardize teratoma assays used to define human pluripotent cell lines. , 2010, Cell stem cell.

[61]  J. Masters,et al.  Detection of Mycoplasma in cell cultures , 2010, Nature Protocols.

[62]  Crystal Jaing,et al.  Viral Nucleic Acids in Live-Attenuated Vaccines: Detection of Minority Variants and an Adventitious Virus , 2010, Journal of Virology.

[63]  R. Lahesmaa,et al.  High-resolution DNA analysis of human embryonic stem cell lines reveals culture-induced copy number changes and loss of heterozygosity , 2010, Nature Biotechnology.

[64]  M. Hurles,et al.  Copy number variation in human health, disease, and evolution. , 2009, Annual review of genomics and human genetics.

[65]  Anika Ashok,et al.  ICH Harmonised Tripartite Guideline , 2009 .

[66]  Moustapha Kassem,et al.  Teratoma formation by human embryonic stem cells is site dependent and enhanced by the presence of Matrigel. , 2009, Stem cells and development.

[67]  R. McKay,et al.  Consensus Guidance for Banking and Supply of Human Embryonic Stem Cell Lines for Research Purposes , 2009, Stem Cell Reviews and Reports.

[68]  S. Morrison,et al.  Efficient tumor formation by single human melanoma cells , 2008, Nature.

[69]  B. Dickens International Society for Stem Cell Research (ISSCR) Guidelines for the Conduct of Human Embryonic Stem Cell Research (December 2006). , 2008, Medicine and law.

[70]  C. Eaves,et al.  Alkaline Phosphatase‐Positive Colony Formation Is a Sensitive, Specific, and Quantitative Indicator of Undifferentiated Human Embryonic Stem Cells , 2008, Stem cells.

[71]  Heli Skottman,et al.  Good manufacturing practice and clinical-grade human embryonic stem cell lines. , 2008, Human molecular genetics.

[72]  C. Croce Oncogenes and cancer. , 2008, The New England journal of medicine.

[73]  C. Simón,et al.  Is teratoma formation in stem cell research a characterization tool or a window to developmental biology? , 2008, Reproductive biomedicine online.

[74]  David E. Vaughan,et al.  Further work on the cryopreservation of articular cartilage with particular reference to the liquidus tracking (LT) method. , 2007, Cryobiology.

[75]  B. Knoppers,et al.  Monetary payments for the procurement of oocytes for stem cell research: In search of ethical and political consistency. , 2007, Stem cell research.

[76]  G. Churchill,et al.  Characterization of human embryonic stem cell lines by the International Stem Cell Initiative , 2007, Nature Biotechnology.

[77]  Ying Liu,et al.  Qualification of Embryonal Carcinoma 2102Ep As a Reference for Human Embryonic Stem Cell Research , 2007, Stem cells.

[78]  G. Stacey,et al.  Cryopreservation and Freeze-Drying Protocols , 1995, Methods in Molecular Biology™.

[79]  B. Knoppers,et al.  Ethics Issues in Stem Cell Research , 2006, Science.

[80]  D. Solter,et al.  From teratocarcinomas to embryonic stem cells and beyond: a history of embryonic stem cell research , 2006, Nature Reviews Genetics.

[81]  J. Loring,et al.  Establishing Standards for the Characterization of Human Embryonic Stem Cell Lines , 2006, Stem cells.

[82]  N. Zaninovic,et al.  Cryopreservation of murine embryos, human spermatozoa and embryonic stem cells using a liquid nitrogen-free, controlled rate freezer. , 2006, Reproductive biomedicine online.

[83]  Michael Balls,et al.  Guidance on Good Cell Culture Practice , 2005, Alternatives to laboratory animals : ATLA.

[84]  M. Kotb,et al.  Human Lymphoid and Myeloid Cell Development in NOD/LtSz-scid IL2Rγnull Mice Engrafted with Mobilized Human Hemopoietic Stem Cells 12 , 2004, The Journal of Immunology.

[85]  G. Fahy,et al.  Improved vitrification solutions based on the predictability of vitrification solution toxicity. , 2004, Cryobiology.

[86]  Mamoru Ito,et al.  NOD/SCID/gamma(c)(null) mouse: an excellent recipient mouse model for engraftment of human cells. , 2002, Blood.

[87]  P. Andrews,et al.  From teratocarcinomas to embryonic stem cells. , 2002, Philosophical transactions of the Royal Society of London. Series B, Biological sciences.

[88]  Alvis Brazma,et al.  On the Importance of Standardisation in Life Sciences , 2001, Bioinform..

[89]  G M Fahy,et al.  Vitrification enhancement by synthetic ice blocking agents. , 2000, Cryobiology.

[90]  Quality of biotechnological products: viral safety evaluation of biotechnology products derived from cell lines of human or animal origin. ICH Harmonised Tripartite Guideline. , 1998, Developments in biological standardization.

[91]  N. Baber,et al.  International conference on harmonisation of technical requirements for registration of pharmaceuticals for human use (ICH). , 1994, British journal of clinical pharmacology.

[92]  Ahmad Husseini,et al.  Canadian standards association , 1993 .

[93]  D. Pegg Viability assays for preserved cells, tissues, and organs. , 1989, Cryobiology.

[94]  P. Addison Human Tissue Act. , 1968, British medical journal.