NIHR GOSH BRC iPSC resource call 2025

The NIHR Great Ormond Street Hospital BRC (GOSH BRC) is a partnership between Great Ormond Street Hospital and the University College London (UCL) Great Ormond Street Institute of Child Health (ICH). GOSH and UCL ICH were first awarded BRC status by the NIHR in 2007. In July 2022, the NIHR awarded BRC status to the GOSH BRC for a fourth term, which runs from December 2022 to November 2027. In this fourth term, as part of a wider national collaboration - a BRC National Paediatric Excellence Initiative has been set up between GOSH BRC and children’s hospitals in Birmingham, Sheffield and Liverpool.

The GOSH BRC’s vision is to transform the health of children, and the adults they will become, by combining cutting edge research methods with world-leading clinical trial expertise, to accelerate discovery of new treatments for children with rare and complex conditions worldwide, delivered through five main research themes: Accelerating Novel Therapies (ANT), Applied Child Health Informatics (ACHI), Gene, Stem and Cellular Therapies (GSCT), Genomic Medicine (GM), Tissue Engineering and Regenerative Medicine (TERM).

The TERM theme aims to pioneer the repair and reconstruction of tissues and organs to treat children with structural malformations, tissue and organ failure, and to improve their life expectancy and quality of life. The theme combines expertise in stem cell biology, iPSC platforms, bioengineering, and surgery to pioneer laboratory-grown organs and develop strategies for tissue and organ restoration.

The ANT theme aims to advance novel precision therapies to clinical translation and eventual licensing as standardised treatments within the NHS and globally. This theme uses iPSCs for preclinical proof-of-concept studies that will eventually facilitate first-in-child UK trials.

Together, the themes invested into the management and development of the GOS-ICH iPSC facility. In the past five years of the BRC, this facility supported a wide range of research groups at the institute, enabling reprogramming of iPSCs from GOSH patient cohort in application to disease modelling and tissue engineering projects.

We plan to support projects at the GOSH and GOS-ICH to generate iPSC lines to facilitate disease modeling and treatments related to childhood diseases, the development of novel cell therapies, and tissue engineering procedures for advanced treatments of malformation and tissue damage.

Through this call we will support the generation of iPSC lines to facilitate disease modeling and treatments related to childhood diseases, the development of novel cell therapies, and tissue engineering procedures for advanced treatments of malformation and tissue damage.

Research coordination support will be available upon request and assessment of capacity and capability. Specifically, support for the recruitment (consenting) of GOSH patients and the collection of tissue collection may be requested (via support of the TERM research coordinator).

The TERM theme already holds ethics approval for “Generation of induced pluripotent stem cell lines to investigate rare disease of childhood” which enables us to obtain tissue for iPSC reprogramming, including; Tissue obtained from routine surgical procedures as part of patient care (including amniotic fluid); skin biopsies; blood; urine; hair follicles; nasal epithelium.

Current methods allow us to reprogram fibroblasts, amniotic fluid stem cells, and mesenchymal cells. We are currently testing our reprogramming methods for other sample types (i.e. hair, blood, urine) and therefore this can be requested as a trial of the methods used in reprogramming currently.

We encourage collaborative submissions from GOS-ICH and GOSH clinical-academic partnerships.

• The applicant must be within the remit of the BRC, i.e., supporting translational research focused on paediatric rare or complex disease.
• Applicants must have an existing employment contract with GOSH or UCL-ICH when they apply, which must be in place for the duration of the award. We encourage collaboration across GOSH and ICH and therefore request that there are Principal Investigators from both GOSH and ICH on each application.
• NIHR funding is formally not allowed to support any research using animals; therefore any project supported through this call must not involve animal work.
• Awardees must acknowledge the NIHR GOSH BRC in any publications and presentations and produce progress reports to the BRC upon request (including one year after the project end date).
• You do not need to have had prior links with the BRC to be eligible to apply. We are keen to receive new ideas from a broad spectrum of research areas across GOSH and ICH within the remit of TERM and ANT themes.

• Complete applications should be sent to brc@gosh.nhs.uk by the deadline of 31 March 2025, 17:00 as a word document
• We will support existing research projects that are registered and approved; new projects will also be considered.
• Applications will be reviewed by the BRC TERM and ANT Leadership Teams.
• Applicants will be notified of the outcome of their application in late April 2025

• Innovative aspects of the proposed project (for which the iPSC lines are intended) and its scientific excellence.
• Feasibility, deliverability of the proposal and, in this call, sample availability:
  • Samples in place: Cells (established cell lines) ready for reprogramming > Active project - easy sample collection from which they can derive fibroblasts > Project with no cells /samples in place.
  • Funding in place: Funding for consumables in place > No resources but important for data generation for planned grant application > No resources and no plans to apply for large funding.
• Likelihood that the project will lead to in future to translational impact for patient benefit.

• For applicants who plan to supply tissue to the iPSC facility under their own REC approval, it is the responsibility of the PI to ensure that appropriate REC approval is in place for the collection and use of tissue for the purpose of iPSC cell line generation and study. Evidence of this will need to be included with the application.
• Applicants will need to agree for the iPSC facility to retain a vial of cultured fibroblasts and 1 vial of each iPSC clone generated from each sample provided as a backup for future use, in cases where clones are generated.
• We also aim to develop a platform to support the establishment of collaborations between GOS-ICH researchers and with external partners to enable sharing of lines from unaffected and affected individuals. With the PI’s permission we will publish plain English summary on the BRC website including the diseases under investigation as part of this programme. Therefore, where samples are obtained using own REC approved ethics, used by PIs who are collecting tissue under their own REC approved projects, need to support the following:
  • Generation of iPSC lines
  • Sharing of iPSC lines with other researchers, including those from commercial entities, and for commercial purposes, for research studies, without a requirement for additional ethical or other approval. It is important that there is no text requiring additional ethical approval for future research studies using the derived cell lines.

Please get in touch with Dr Marco Pellegrini (marco.pellegrini@ucl.ac.uk) to discuss the needs of your project and if you have any specific questions about the methods available.

For any other queries, please contact Valerie Karaluka (brc@gosh.nhs.uk)