Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Age-Related Macular Degeneration

Background: Dry age-related macular degeneration (AMD) is one of the leading causes of vision loss in older patients. The macula accumulates drusen with loss of retinal pigment epithelial cells and photoreceptors. Abnormal subretinal neovascularization is absent. There is no effective drug therapy for dry AMD and a large proportion of patients progress to legal blindness from macular atrophy. The Stem Cell Ophthalmology Treatment Study (SCOTS) was conducted to assess the effect of bone marrow-derived stem cells (BMSCs) on dry AMD and other retinal and optic nerve diseases. Methods: Thirty-two eyes were treated with BMSC per the protocols in SCOTS. Provision of BMSCs in Arm 1 was via retrobulbar (RB), sub-tenons (ST) and intravenous (IV); Arm 2 via intravitreal, RB, ST and IV; Arm 3 via subretinal and IV. Patient age averaged 78 years old and ranged from 69 to 90. Visual acuity preoperatively ranged from counting fingers to 20/50-2 with an average preoperative LogMAR of 1.125. Results: Following treatment, 20 of 32 (63%) of eyes experienced improvement in visual acuity averaging 27.6% on LogMAR and ranging from 2.5% to 44.6%. The mean improvement in LogMAR was 0.963 with a standard deviation (SD) of 0.42. The visual acuity remained stable in 34% of treated eyes. One eye continued to worsen as a consequence of disease progression. The results showed high statistical significance with p ≤ 0.001. The procedures were conducted safely, and no complications were observed. Conclusions: Treatment of dry AMD with BMSC using the protocols developed in the SCOTS clinical trial has shown statistically significant clinical benefit improving visual acuity and potentially delaying visual loss in the disease.