Treatment of traumatic optic neuropathy using human placenta-derived mesenchymal stem cells in Asian patients

Aim: To assess the safety and feasibility of subtenon transplantation of human placenta-derived mesenchymal stem cells (hPMSCs) in Asian patients with traumatic optic neuropathy. Materials & methods: The survival of retinal ganglion cells in the rat retina was evaluated by monitoring the expression of Tuj1 and Gfap after optic nerve compression. Based on the preclinical data, we conducted a Phase I, open label, single center, nonrandomized clinical trial in four Asian traumatic optic neuropathy patients. The safety and ophthalmologic changes were evaluated. Results: The levels of Tuj1 and Gfap expression were significantly increased in the hPMSC treatment group compared with the sham group, suggesting a protective effect of hPMSCs on the optic nerve and retinal ganglion cells. There was no evidence of adverse proliferation, tumorigenicity, severe inflammation or other serious issues during the 12-month follow-up period. Visual acuity improved in all four patients. Conclusion: The results suggested that hPMSCs are safe and have potential utility in regenerative medicine. Clinical trial registration number: 20150196587 (Korean FDA), 2015-07-123-054 (IRB).