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Corneal Stem Cell Reconstruction Project

This project is aimed at restoring vision to people suffering with severe forms of corneal disease that has left them blind with little to no hope of seeing again. Corneal stem cell deficiency can be caused by infections, immune diseases, prolonged topical drug use, trauma and many other causes. For such patients, standard corneal transplants are unsuccessful as their corneas are so diseased that they cannot accept grafts from other humans despite taking anti-rejection medicines. Even if the graft takes and is not rejected, often the clarity is limited as the surface of the graft becomes opaque in time if there are few healthy stem cells. The major goal of our studies has been to provide an alternative to the corneal transplant by using the patient’s own tissue(s) to regenerate the blind cornea. For people who have corneal stem cell deficiency in one eye, stem cells from the other eye can be grown in the laboratory and used to rebuild the cornea. For people who have both corneas affected by the disease, stem cells from the mouth region can be used to renew the corneal surface and restore vision.

Over the past year, we have made breakthrough progress in further developing this technology and bringing it closer to patient use:

1. In the laboratory, we continue to optimize and standardize culture conditions for cultivating stem cells and “coaxing” them to differentiate into cornea cells using human “feeder cells” derived from human skin. This process has reached a stage which now employs all human-derived products, including human serum, human growth factors, and human feeder cells from one batch and thus aims at growing the cells under most consistent and ‘patient-friendly’ conditions. We are still in the process of delineating the best ‘release criteria’ that represent the parameters that determine when the stem cell sheets are most optimal for corneal transplantation. In order to do so, we are investigating different imaging modalities and live-cell dyes to monitor cell growth before transplantation.

2. We have also made excellent progress in establishing collaboration between the Department of Ophthalmology and the Center for Human Cell Therapy (CHCT) at the Immune Disease Institute and the Connell O'Reilly Cell Manipulation Core Facility at the Dana-Farber Cancer Institute. The collaborators at the Center for Human Cell Therapy will be performing the “translational phase” of the project. They will perform the validation experiments, toxicological and cell viability testing. Their part will be crucial for going to the “clinical phase” which will take place in the O'Reilly Cell Manipulation Core Facility at Dana-Farber.

The next step in bringing this method to patients is to prepare an application to the FDA requesting permission to start applying these technologies to humans. Our collaborators will work with us to provide the necessary skill set and facilities to perform such studies in order to prepare the FDA application and ultimately manufacture the stem cell constructs in the Good Manufacturing Practice Laboratories across the U.S.