Unmet Need
Fuchs’ dystrophy, a progressive, hereditary disease of the cornea, is the leading cause of corneal transplants both in the US and worldwide. The disease results in the dysregulation and destruction of corneal endothelial cells which are responsible for keeping the eye properly hydrated. As the disease progresses, patients will experience blurred vision eventually culminating in blindness. The damaged corneal endothelial cells do not heal and donor-dependent, transplantation surgery is the only cure for Fuchs’ and other corneal endothelial diseases. Unfortunately, transplanted corneas fail as frequently as 10-20% of time resulting in multiple procedures despite the eye’s status as an immune privileged organ and anti-rejection eye drops. Thus, there is a need for transplantable corneal endothelial cells that do not trigger a host immune response.
Technology Overview
Johns Hopkins researchers have discovered a method of generating corneal endothelial cells from a patient’s own blood cells, dramatically limiting the potential for an immune response while extending treatment to patients who can’t find a suitable donor. Blood cells taken from the patient are first transformed into induced pluripotent stem cells and subsequently differentiated into corneal endothelial cells over a 20-day period. These stem cell-derived corneal endothelial cells have the same physical morphology and shape as authentic human corneal endothelial cells. Moreover, mass spectrometry proteomics and gene expression analysis suggest that the lab-grown cells and human derived cells are >90% identical. It is important to note that pluripotency markers are only expressed at basal levels in the stem cell-derived corneal endothelial cells indicating the cells have terminally differentiated and are not potentially oncogenic. Successful implementation of this technology will bring personalized, patient-specific approaches to corneal transplantation and reduce the dependency on donor corneas as well as decreasing the incidence rate of immune rejection.
Stage of Development
In vitro and animal model data are available.
Publications
https://www.cell.com/stem-cell-reports/fulltext/S2213-6711(21)00374-X#secsectitle0010
Invest Ophthalmol Vis Sci. 2018 May 1;59(6):2437-2444.
