Efficacy and Safety of Autologous Bone Marrow Mesenchymal Stem Cell Transplantation in Patients with Diabetic Retinopathy.

ABSTRACT

BACKGROUND/AIMS: The treatment options for diabetic retinopathy (DR) are limited. Mesenchymal stem cells (MSCs) are a promising treatment option for diabetes and its complications. In this pilot clinical trial, we evaluated the safety and efficacy of intravenous autologous bone marrow MSCs (ABMSC) for the treatment of DR. METHODS: In total, 34 eyes with non-proliferative or proliferative DR (NPDR, n = 19; PDR, n = 15) from 17 patients were analyzed. Treatment involved one intravenous infusion of 3 × 106/kg ABSMCs. The patients' vital signs were monitored, along with immune and allergic reactions. Treatment efficacy was evaluated via measurements of the following parameters at baseline, and at 1, 3, and 6 months after treatment the levels of fasting blood glucose (FBG), Hemoglobin A1C (HbA1C), interleukin-6 (IL-6), and hypersensitive C-reactive protein (CRP); best corrected visual acuity (BCVA); and central macular and subfield thickness (via optical computed tomography). RESULTS: ABMSC infusion led to a significant decrease in FBG and CRP levels (P < 0.05). There were no significant differences in HbA1C or IL-6 levels. Sub-group analysis revealed that only eyes in the NPDR group had the macular thickness reductions and a significant improvement in BCVA from baseline (P = 0.006 at 3 months and 0.027 at 6 months), while those in the PDR group did not. There were no acute reactions during the treatment or severe adverse events during the follow-up period. CONCLUSION: ABSMCs are a potentially safe and effective treatment option for DR, and the optimum therapeutic window appears to be during the NPDR stage.