Corneal Epithelial Stem Cell Topical Therapy in DED

Article: TTUHSC Technology Leads Successful Clinical Trial to Treat Dry Eye Disease
Source: Texas Tech University Health Sciences Center, School of Pharmacy
Published: September 29, 2021

Dry eye disease is estimated to affect up to half of the U.S. population. Depending on the underlying etiology, a wide range of therapies are available. Pharmaceutical scientists, along with clinical colleagues, are exploring a topical delivery of corneal epithelial stem cells, located and obtained solely at the limbus, as a potentially safer and more effective treatment option. They report excellent results in the first stage of clinical trials. Collaborating with a Good Manufacturing Practice (GMP) facility, they isolated and expanded the human corneal stem cells, obtained from donor tissues from an eye bank, to derive a topically applied medicine in this case. The study recruited 17 participants (34 eyes) suffering from advanced dry eye disease, each of whom have documented attempts to treat their dry eyes using between 6 and 14 conventional dry eye therapies. These participants also qualified for the trial based on a score greater than or equal to 14 on the Standardized Patient Evaluation of Eye Dryness (SPEED™) questionnaire and a score greater than or equal to 40 on the Ocular Surface Disease Index (OSDI©). The lead researcher of the study remarks, “Nothing was helping them; they were in a very severe condition. Some of them could only go outside in the dark because their inflammation was so bad that sunlight would burn their eyes.”

During the trial, each patient self-administered the topical corneal epithelial stem cell-derived product four times daily in both eyes for 12 weeks. Compared to baseline, there was a 23% improvement in SPEED™ scores and a 17% improvement in OSDI© scores (but not statistically significant). An additional dry eye assessment questionnaire (University of North Carolina Dry Eye Management Scale) showed a 14% score improvement. Objective measurements included best-corrected visual acuity (BCVA), corneal topography, and tear film osmolarity, none of which changed significantly in the 12 weeks. None of the study participants reported any adverse effects and, as the authors are keen to note, all asked to resume use of the drops after the study. The project lead speculates that the success of the therapy is likely because it both reduces inflammation and regenerates damaged tissue, as stem cells would. They conclude that topical corneal epithelial stem cell-derived supernatant that can be self-administered by the patient shows promise at improving patient symptoms and quality of life in severe DED that is unresponsive to conventional therapies. They next plan a double-blinded study and have filed patents for their product.

My rating of this study: ⭐⭐⭐

Rush SW, Chain J, Das H, et al. "Corneal Epithelial Stem Cell Supernatant in the Treatment of Severe Dry Eye Disease: A Pilot Study." Journal of Clinical Ophthalmology.  15:3097-3107. 16 July 2021. https://doi.org/10.2147/OPTH.S322079