Preclinical study of novel human allogeneic adipose tissue-derived mesenchymal stem cell sheets toward a first-in-human clinical trial for myopic chorioretinal atrophy.

Several human studies show positive signals, while research methods and sample sizes continue to develop.

• Level A · Stronger Clinical Evidence
• Level B · Emerging clinical evidence with positive signals
• Level C · Early human research exploring benefits
• Level D · Scientific groundwork from lab and animal studies
• Emerging · Emerging topic under active research

Abstract (original English)

Mesenchymal stem cells may have neuroprotective and tissue regenerative capabilities and the potential to rescue retinal degeneration in chorioretinal diseases including myopic chorioretinal atrophy. Transplantation of human (allogeneic) adipose tissue-derived mesenchymal stem cell (adMSC) suspensions has been clinically conducted to treat retinal degenerative diseases. However, serious side effects including proliferative vitreoretinopathy and epiretinal membrane formation have been reported. PharmaBio Corporation fabricated novel adMSC sheets with a Bruch's membrane-like structure using our original method, potentially overcoming these problems. We evaluated the characteristics of newly developed adMSC sheets named PAL-222 and assessed their safety and efficacy in rats with congenital retinal degeneration (RCS rats) to obtain the proof-of-concept for the first-in-human clinical trial for myopic chorioretinal atrophy. We measured the viability of cells obtained from PAL-222, examined cell surface antigens by flow cytometry, measured the vascular endothelial growth factor (VEGF) and pigment epithelium-derived factor (PEDF) secretory ability, examined the expression of types I and IV collagen and elastin by immunostaining. We performed a transwell in vitro migration assay to evaluate durability and similarity to retinal pigment epithelium (RPE) and checked in vitro tumorigenicit

Evidence level

Several human studies show positive signals, while research methods and sample sizes continue to develop.

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