Abstract: Te Stem Cell Ophthalmology Treatment Study (SCOTS) is currently the largest-scale stem cell ophthalmology trial registered at ClinicalTrials.gov (identifer: NCT01920867). SCOTS utilizes autologous bone marrow-derived stem cells (BMSCs) to treat optic nerve and retinal diseases. Treatment approaches include a combination of retrobulbar, subtenon, intravitreal, intra-optic nerve, subretinal, and intravenous injection of autologous BMSCs according to the nature of the disease, the degree of visual loss, and any risk factors related to the treatments. Patients with Leber’s hereditary optic neuropathy had visual acuity gains on the Early Treatment Diabetic Retinopathy Study (ETDRS) of up to 35 letters and Snellen acuity improvements from hand motion to 20/200 and from counting fngers to 20/100. Visual feld improvements were noted. Macular and optic nerve head nerve fber layer typically thickened. No serious complications were seen. Te increases in visual acuity obtained in our study were encouraging and suggest that the use of autologous BMSCs as provided in SCOTS for ophthalmologic mitochondrial diseases including Leber’s hereditary optic neuropathy may be a viable treatment option.

Key words: nerve regeneration, Leber’s hereditary optic neuropathy, mitochondrial disease, optic neuropathy, bone marrow derived stem cells, blindness, visual loss, neural regeneration