Periodic Reporting for period 3 - hPSCreg (Human Puripotent Stem Cell Registry)
Période du rapport: 2020-01-01 au 2021-12-31
Human pluripotent stem cells (hPSC) differentiate in all cell types of the human body, expand without limit and are genetic surrogates of their donors. These characteristics make them enormously valuable for cell therapy, for disease and tissue modelling testing of drugs. Human embryonic stem cell (hESC) lines are derived from early human embryos raising ethical issues manifested in diverse regulatory frameworks globally and within the EU. Human induced pluripotent stem cells (hiPSC) can be generated from any somatic cell. Both cell types are extensively used in research and in clinical trials. The hPSCreg provides information about globally available hPSC – lines, their ethical provenance, their biological quality and their availability and use.
The hPSCreg developed into an invaluable resource for developing and promoting standards and guidelines and to implement FAIR data principles. hPSCreg application of validated and transparent standard requirements aupports the need for reproducible research. In addition, the Registry reduces risks in the field by allowing comparability and reproducibility, as well as avoiding waste of resources by providing access to existing hPSC-lines worldwide.
The hPSCreg is driven by the needs to gather and make available detailed information on the different hPSC lines avoiding needless creation of new cell lines. The Registry operates through an internet portal.
The operations are divided into the main concept areas (i) acquisition, registration and qualification of information, (ii) communication, dissemination and harmonization, (iii) technical implementation, governance and project management. The continuous work areas are (1) Provision of validated ethics information for each cell line, (2) Provision of validated scientific information for each cell line, (3) Provision of information on clinical application of hPSC lines, (4) Strengthen international reach and community interaction as well as acceptance of quality and data standards and integration into mechanisms to assure reproducibility in research, (5) Maintaining and improving a stable state of the art back- and frontend and (6) Establish and nurture synergistic interaction with key infrastructures such as hPSC – banks and registries, (7) Maintain transparent management structure.
The hPSCreg developed into an invaluable resource for developing and promoting standards and guidelines and to implement FAIR data principles. hPSCreg application of validated and transparent standard requirements aupports the need for reproducible research. In addition, the Registry reduces risks in the field by allowing comparability and reproducibility, as well as avoiding waste of resources by providing access to existing hPSC-lines worldwide.
The hPSCreg is driven by the needs to gather and make available detailed information on the different hPSC lines avoiding needless creation of new cell lines. The Registry operates through an internet portal.
The operations are divided into the main concept areas (i) acquisition, registration and qualification of information, (ii) communication, dissemination and harmonization, (iii) technical implementation, governance and project management. The continuous work areas are (1) Provision of validated ethics information for each cell line, (2) Provision of validated scientific information for each cell line, (3) Provision of information on clinical application of hPSC lines, (4) Strengthen international reach and community interaction as well as acceptance of quality and data standards and integration into mechanisms to assure reproducibility in research, (5) Maintaining and improving a stable state of the art back- and frontend and (6) Establish and nurture synergistic interaction with key infrastructures such as hPSC – banks and registries, (7) Maintain transparent management structure.
The major work areas are: (1) maintaining and improving technical and scientific functionality, (2) development of Registry content, (3) dissemination and (4) management and governance.
1. The Registry's unique ethics data model was finalized and a version for data output and searchability developed. An updated interface for ethics data comparison and searching was implemented. To ease data deposition, API were developed and are available. The unique persistent identifier for hPSC – lines was published. Validation and certification of lines was moved from a manual to a mostly automated process, reducing error rates. To improve searching and filtering, a portal design was developed and is being tested with stakeholder feedback.
2. Cell line registration is a continuous task. The registration data model was further improved and mandatory data elements redefined. The number of registered hESC-Lines increased within the period by 5783 iPSC and 305 hESC - lines. The number of registered projects increased to 220. Clinical use of hPSC will be the next revolution in cell therapy. hPSCreg implemented a dedicated database for clinical trials. 96 clinical studies registered: hESC (n=48), hiPSC (n=45) and parthenotic PSC (n=3).
3. The Registry has further developed collaborations with international stakeholder organizations and Banks in Europe, the USA and Asia. Dissemination was mainly through the hPSCreg Portal, were open access is provided to hPSCreg content, governance documents, best practice and other procedural documents. The Committee of National Representatives (CNR), with currently 33 members from EU and Non-EU countries with registered lines in hPSCreg, is the main advisory and interaction group with key stakeholders, also with respect to following-up of changing regulatory and legal frameworks. The dissemination is reflected in more than 51 presentations at conferences, workshops, webinars and training events, as well as 18 publications in peer reviewed international journals. hPSCreg is involved in the EU-funded Projects EJP-RD and EuroGCT.
4. The Registry team maintained an accurate, safe and carefully constructed database (backend) and portal (front end) as a continuous task, which fully complies with GDPR. An adequate management structure was implemented and relevant documentation and Registry - related administrative documents are publicly accessible. FAIR data principles were further improved. The Registry is supported by an Advisory Board and the CNR.
1. The Registry's unique ethics data model was finalized and a version for data output and searchability developed. An updated interface for ethics data comparison and searching was implemented. To ease data deposition, API were developed and are available. The unique persistent identifier for hPSC – lines was published. Validation and certification of lines was moved from a manual to a mostly automated process, reducing error rates. To improve searching and filtering, a portal design was developed and is being tested with stakeholder feedback.
2. Cell line registration is a continuous task. The registration data model was further improved and mandatory data elements redefined. The number of registered hESC-Lines increased within the period by 5783 iPSC and 305 hESC - lines. The number of registered projects increased to 220. Clinical use of hPSC will be the next revolution in cell therapy. hPSCreg implemented a dedicated database for clinical trials. 96 clinical studies registered: hESC (n=48), hiPSC (n=45) and parthenotic PSC (n=3).
3. The Registry has further developed collaborations with international stakeholder organizations and Banks in Europe, the USA and Asia. Dissemination was mainly through the hPSCreg Portal, were open access is provided to hPSCreg content, governance documents, best practice and other procedural documents. The Committee of National Representatives (CNR), with currently 33 members from EU and Non-EU countries with registered lines in hPSCreg, is the main advisory and interaction group with key stakeholders, also with respect to following-up of changing regulatory and legal frameworks. The dissemination is reflected in more than 51 presentations at conferences, workshops, webinars and training events, as well as 18 publications in peer reviewed international journals. hPSCreg is involved in the EU-funded Projects EJP-RD and EuroGCT.
4. The Registry team maintained an accurate, safe and carefully constructed database (backend) and portal (front end) as a continuous task, which fully complies with GDPR. An adequate management structure was implemented and relevant documentation and Registry - related administrative documents are publicly accessible. FAIR data principles were further improved. The Registry is supported by an Advisory Board and the CNR.
hPSCreg provides companies and researchers with a tool to help reduce legal and regulatory risks to commercial R&D by cell selection through availability of qualified ethics and scientific data. The hPSCreg data model also serves as basis for EBiSC and related resources. In addition, hPSCreg helps to set standards in hPSC – data elements, use of standard language, application of biological quality standards, implementation of FAIR data principles, and supports banks and registries to link and share data. Through training events and webinar presentations hPSCreg supports use of the named standards and provides rational and justification to register lines in hPSCreg as well as to use hPSCreg to minimize research risks.
The Registry provides direct access to ethics provenance information. This is unique and allows assessment of suitability from an ethical point of view. The ethics assessment of the hPSC provenance has been developed by hPSCreg and is open for reuse to mitigate risks associated with missing or deficient consent. In addition, hPSCreg validates the scientific quality of hPSC – lines and issues certificates, which verify that a line adheres ethically and scientifically to the highest standards, based on the submitted data. These certificates are required for funding of projects involving hPSC - lines for example by the European Commission. hPSCreg serves as a reviewer for publication in the journal Stem Cell Research – Lab Resource by validating submitted data on hPSC – lines.
Stem cell technology has significant potential to improve human health with huge economic potential. In providing an international coordination activity and best practice, hPSCreg impacts stem cell-based industries and increase their competiveness by supporting standards and harmonization, comparability and selection of the most effective protocols and cell lines. Implementation of hPSCreg’s clinical trial database serves as a unique resource of knowledge and information on ongoing therapeutic research in the field.
hPSCreg's advantage for sustainability is based on a comprehensive, inclusive database hosting the world’s largest collection of validated hPSC lines. Its societal implications lies in the provision of transparent oversight of ethically controversial research, providing control and assurance of ethical conduct to European and global citizens. Te socio economic impat´ct lies in setting and pushing forward quality standards in the field, which is prerequisite for reproducibility of research.
The Registry provides direct access to ethics provenance information. This is unique and allows assessment of suitability from an ethical point of view. The ethics assessment of the hPSC provenance has been developed by hPSCreg and is open for reuse to mitigate risks associated with missing or deficient consent. In addition, hPSCreg validates the scientific quality of hPSC – lines and issues certificates, which verify that a line adheres ethically and scientifically to the highest standards, based on the submitted data. These certificates are required for funding of projects involving hPSC - lines for example by the European Commission. hPSCreg serves as a reviewer for publication in the journal Stem Cell Research – Lab Resource by validating submitted data on hPSC – lines.
Stem cell technology has significant potential to improve human health with huge economic potential. In providing an international coordination activity and best practice, hPSCreg impacts stem cell-based industries and increase their competiveness by supporting standards and harmonization, comparability and selection of the most effective protocols and cell lines. Implementation of hPSCreg’s clinical trial database serves as a unique resource of knowledge and information on ongoing therapeutic research in the field.
hPSCreg's advantage for sustainability is based on a comprehensive, inclusive database hosting the world’s largest collection of validated hPSC lines. Its societal implications lies in the provision of transparent oversight of ethically controversial research, providing control and assurance of ethical conduct to European and global citizens. Te socio economic impat´ct lies in setting and pushing forward quality standards in the field, which is prerequisite for reproducibility of research.