Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2023 Aug 8;18(8):1592-1598.
doi: 10.1016/j.stemcr.2023.03.005. Epub 2023 Apr 6.

History and current status of clinical studies using human pluripotent stem cells

Sabine Kobold  1 , Nils Bultjer  1 , Glyn Stacey  2 , Sabine C Mueller  1 , Andreas Kurtz  3 , Nancy Mah  4
Affiliations
Review

History and current status of clinical studies using human pluripotent stem cells

Sabine Kobold et al. Stem Cell Reports. .

Abstract

The Human Pluripotent Stem Cell Registry established a database of clinical studies using human pluripotent stem cells (PSCs) as starting material for cell therapies. Since 2018, we have observed a switch toward human induced pluripotent stem cells (iPSCs) from human embryonic stem cells. However, rather than using iPSCs for personalized medicines, allogeneic approaches dominate. Most treatments target ophthalmopathies, and genetically modified iPSCs are used to generate tailored cells. We observe a lack of standardization and transparency about the PSCs lines used, characterization of the PSC-derived cells, and the preclinical models and assays applied to show efficacy and safety.

PubMed Disclaimer

Conflict of interest statement

Conflict of interests The authors declare no competing interests.

Figures

Figure 1
Figure 1
Temporal distribution of studies using ESCs, iPSCs, somatic cell nuclear transfer (SCNT) cells, and parthenotic PSCs (pPSCs) with start dates within the period of January 1, 2011–December 13, 2022 Data are aggregated into 2-year bins.
Figure 2
Figure 2
Sponsor type per country (ISO 3166-1 three-letter codes)
Figure 3
Figure 3
Heatmap showing frequencies of PSC-derived cell types used in clinical studies for the last decade
See this image and copyright information in PMC

References

    1. Cornetta K., Bonamino M., Mahlangu J., Mingozzi F., Rangarajan S., Rao J. Gene therapy access: global challenges, opportunities, and views from Brazil, South Africa, and India. Mol. Ther. 2022;30:2122–2129. doi: 10.1016/j.ymthe.2022.04.002. - DOI - PMC - PubMed
    1. Creasey A.A., Stacey G., Bharti K., Sato Y., Lubiniecki A. A strategic road map to filing a Biologics License Application for a pluripotent stem cell derived therapeutic product. Biologicals. 2019;59:68–71. doi: 10.1016/j.biologicals.2019.03.007. - DOI - PMC - PubMed
    1. DeVito N.J., Bacon S., Goldacre B. Compliance with legal requirement to report clinical trial results on ClinicalTrials.gov: a cohort study. Lancet. 2020;395:361–369. doi: 10.1016/S0140-6736(19)33220-9. - DOI - PubMed
    1. Enosawa S. Clinical trials of stem cell therapy in Japan: the decade of progress under the national program. J. Clin. Med. 2022;11:7030. doi: 10.3390/jcm11237030. - DOI - PMC - PubMed
    1. Ginn S.L., Amaya A.K., Alexander I.E., Edelstein M., Abedi M.R. Gene therapy clinical trials worldwide to 2017: an update. J. Gene Med. 2018;20:e3015. doi: 10.1002/jgm.3015. - DOI - PubMed

Publication types

MeSH terms

LinkOut - more resources