Lessons from human teratomas to guide development of safe stem cell therapies

Justine J. Cunningham, Thomas Ulbright, Martin F. Pera, Leendert H J Looijenga

Research output: Contribution to journalArticle

103 Citations (Scopus)

Abstract

The potential for the formation of teratomas or other neoplasms is a major safety roadblock to clinical application of pluripotent stem cell therapies. Preclinical assessment of the risk of tumor formation in this context poses considerable scientific and regulatory challenges, especially because animal xenograft models may not properly reflect the long-term tumorigenic potential of human cells. A better understanding of the biology of spontaneously occurring teratomas and related tumors in humans can help to guide efforts to assess and minimize the potential hazards of embryonic stem cell or induced pluripotent stem cell therapeutics. Here we review the features of teratomas derived experimentally from human pluripotent stem cells and argue that they most closely resemble spontaneous benign teratomas that occur early in both mouse and human life. The natural history and pathology of these spontaneously occurring teratomas provide important clues for preclinical safety assessment and patient monitoring in trials of stem cell therapies.

Original languageEnglish
Pages (from-to)849-857
Number of pages9
JournalNature Biotechnology
Volume30
Issue number9
DOIs
StatePublished - Sep 2012

Fingerprint

Teratoma
Cell- and Tissue-Based Therapy
Stem cells
Stem Cells
Pluripotent Stem Cells
Tumors
Safety
Patient monitoring
Induced Pluripotent Stem Cells
Neoplasms
Physiologic Monitoring
Pathology
Embryonic Stem Cells
Natural History
Heterografts
Hazards
Animals
Animal Models
Cells

ASJC Scopus subject areas

  • Applied Microbiology and Biotechnology
  • Biotechnology
  • Molecular Medicine
  • Bioengineering
  • Biomedical Engineering

Cite this

Lessons from human teratomas to guide development of safe stem cell therapies. / Cunningham, Justine J.; Ulbright, Thomas; Pera, Martin F.; Looijenga, Leendert H J.

In: Nature Biotechnology, Vol. 30, No. 9, 09.2012, p. 849-857.

Research output: Contribution to journalArticle

Cunningham, Justine J. ; Ulbright, Thomas ; Pera, Martin F. ; Looijenga, Leendert H J. / Lessons from human teratomas to guide development of safe stem cell therapies. In: Nature Biotechnology. 2012 ; Vol. 30, No. 9. pp. 849-857.
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