Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow Derived Stem Cells in the treatment of Non-arteritic Ischemic Optic Neuropathy  (NAION)

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


Ten patients with bilateral visual loss due to sequential non-arteritic ischemic optic neuropathy (NAION) underwent autologous bone marrow derived stem cell therapy within the Stem Cell Ophthalmology Treatment Study (SCOTS).  SCOTS is an Institutional Review Board approved clinical study utilizing autologous Bone Marrow Derived Stem Cells (BMSC) in the treatment of optic nerve and retinal diseases that meet inclusion criteria.


The average age of the patients treated was 69.8 years.  The average duration of
visual loss in eyes treated was 9.8 years and ranged from 1 to 35 years. Affected
eyes were treated with either retrobulbar, subtenons and intravenous BMSC or, following vitrectomy, intraoptic nerve and intravenous BMSC.  The primary outcome  was visual acuity as measured by Snellen or converted to LogMAR.


Following therapy in SCOTS, 80% of patients experienced improvement in Snellen binocular vision (p=0.029) with 20% remaining stable; 73.6 % of eyes  treated gained vision(p=0.019) and 15.9% remained stable in the post-operative period. There was an average of 3.53 Snellen lines of vision improvement per eye  with an average 22.74% and maximum 83.3% improvement in LogMAR acuity per eye. The average LogMAR change in treated eyes was a gain of 0.364 (p=0.0089). Improvements typically manifested no later than 6 months post procedure.


The use of BMSC in the Stem Cell Ophthalmology Treatment Study achieved meaningful visual improvements in a significant percentage of the NAION patients reported.  Improvements typically manifested no later than 6 months post procedure.   Duration of visual loss did not appear to affect the ability of  the eyes to respond to treatment.  Possible mechanisms by which visual improvement occurred may include BMSC paracrine secretion of proteins and hormones, transfer of mitochondria, release of messenger RNA or other compounds via exosomes or micro vesicles and neuronal transdifferentiation of the stem cells.