Stem Cell Ophthalmology Treatment Study (SCOTS): bone marrow derived stem cells in the treatment of Dominant Optic Atrophy.

Background: We report the results of 6 patients with Dominant Optic Atrophy (DOA) who met inclusion criteria and were treated in the Stem Cell Ophthalmology Treatment Study (SCOTS). SCOTS/SCOTS 2 is an Institutional Review Board approved and NIH registered (NCT03011541) clinical study that uses autologous bone marrow derived stem cells (BMSC) in the treatment of optic nerve and retinal disease.

Methods: This is an open label, non-randomized clinical study using natural history of the disease as the comparator. BMSC were separated from aspirated autologous bone marrow with minimal manipulation using an FDA cleared Class II medical device. Patients were treated with combinations of retrobulbar, subtenons, intravitreal or subretinal placement of BMSC followed by intravenous injection of BMSC depending on the arm of the study chosen. There were no surgical complications.

Results: Of the patients treated, 83.3% (5 of 6 patients) experienced visual improvements and in all of these cases both eyes improved. Ten eyes or 83.3% experienced gains in visual acuity with a median improvement of 2.125 Snellen lines, or approximately 10.63 letters. Two eyes were considered unchanged compared to longstanding measurements. Using LogMAR, the average improvement in vision for all eyes was 29.5%. The averagevisual acuity increasein eyes that improved was 33.3%. Findings were statistically significant with P<0.001.

Conclusions: Using autologous BMSC per protocols developed in the SCOTS/SCOTS 2 clinical studies resulted in statistically significant visual acuity improvements in patients with DOA or Kjers Optic Neuropathy. Improvements occurred in 83.3% of eyes and averaged 29.5%. Mitochondrial transfer and neuroprotective exosome secretions from the BMSC may have been key to the improvements observed in this mitochondrial disease.