Stem Cell Ophthalmology Treatment Study (SCOTS): bone marrow-derived stem cells in the treatment of Leber's hereditary optic neuropathy

Jeffrey N Weiss, Steven Levy, Susan C Benes, Jeffrey N Weiss, Steven Levy, Susan C Benes

Abstract

The Stem Cell Ophthalmology Treatment Study (SCOTS) is currently the largest-scale stem cell ophthalmology trial registered at ClinicalTrials.gov (identifier: 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 fingers to 20/100. Visual field improvements were noted. Macular and optic nerve head nerve fiber layer typically thickened. No serious complications were seen. The 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.

Keywords: Leber's hereditary optic neuropathy; blindness; bone marrow derived stem cells; mitochondrial disease; nerve regeneration; neural regeneration; optic neuropathy; visual loss.

Conflict of interest statement

Conflicts of Interest: None declared.

Figures

Figure 1
Figure 1
Optical coherence tomography of thinned macula in patient 2 (August 19, 2013).
Figure 2
Figure 2
Optical coherence tomography of thinned retinal nerve fiber layer in patient 2 (August 19, 2013).
Figure 3
Figure 3
Optical coherence tomography of thickened retinal nerve fiber layer in patient 2 (April 29, 2015).
Figure 4
Figure 4
Optical coherence tomography of thickened macula in patient 2 (April 29, 2015).
Figure 5
Figure 5
Early Treatment Diabetic Retinopathy Study (ETDRS) reported improvement in Leber's hereditary optic neuropathy genetic type and Snellen visual acuities. L: Left; R: right; NLP: no light perception; CF: counting fingers; HM: hand motion.

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Source: PubMed

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