Induced pluripotent stem-cell-derived corneal epithelium for transplant surgery: a single-arm, open-label, first-in-human interventional study in Japan
2024

First Human Study of Stem Cell-Derived Corneal Transplant

Sample size: 4 publication Evidence: low

Author Information

Author(s): Soma Takeshi MD, Oie Yoshinori MD, Takayanagi Hiroshi MS, Matsubara Shoko BA, Yamada Tomomi PhD, Nomura Masaki PhD, Yoshinaga Yu MD, Maruyama Kazuichi MD, Watanabe Atsushi MD, Takashima Kayo PhD, Mao Zaixing PhD, Quantock Andrew J Prof PhD, Hayashi Ryuhei PhD, Nishida Kohji Prof MD

Primary Institution: Department of Ophthalmology, Graduate School of Medicine, Osaka University, Suita, Osaka, Japan

Hypothesis

Can induced pluripotent stem cell-derived corneal epithelial cell sheets effectively treat limbal stem cell deficiency in humans?

Conclusion

The transplantation of stem cell-derived corneal epithelial cell sheets was found to be safe and showed positive clinical outcomes in all patients over a two-year period.

Supporting Evidence

  • All four patients showed improvement in clinical stage and visual acuity after the procedure.
  • No serious adverse events occurred during the two-year follow-up.
  • Patients reported improved quality of life and reduced corneal opacification.
  • Adverse events were mostly mild and manageable.

Takeaway

Doctors used special cells from stem cells to help four people see better after they had serious eye problems, and it worked well without causing major issues.

Methodology

This was a non-randomised, single-arm clinical study involving four patients with limbal stem cell deficiency, where iPSC-derived corneal epithelial cell sheets were transplanted and monitored for safety and efficacy over 52 weeks.

Potential Biases

Potential for selection bias due to the non-randomised design.

Limitations

The study had a small sample size, limiting the generalizability of the results.

Participant Demographics

Four patients, all self-identified as ethnic Japanese, aged between 39 and 72 years.

Digital Object Identifier (DOI)

10.1016/S0140-6736(24)01764-1

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