• Beyond Corneal Transplants: Endothelial Cell Replacement

    In an overview of current cell therapy research, Jeffrey L. Goldberg, MD, PhD, discussed a novel method of transplanting corneal endothelial cells that may eventually solve some of the challenges inherent in corneal transplants. He spoke during “The Artificial Eye: 2020 and the Dawn of Innovation” (Sym35V).

    Magnetic nanoparticles to the rescue. In this method, human corneal endothelial cells (HCECs) are expanded in culture, labeled with magnetic nanoparticles, and injected into the anterior chamber. In animal studies, Dr. Goldberg and his colleagues evaluated the ability of these transplanted HCECs to reduce corneal edema. They found that corneal transparency was restored without detectable toxicity or adverse effects on intraocular pressure.1

    In human studies, after the cells are injected, the patient wears a magnetic eye patch for at least an hour, or perhaps overnight, to “facilitate the localization and integration of these HCECs into the endothelial layer,” Dr. Goldberg said.

    Rationale. The premise of this approach is that it will be able to overcome key barriers to cell therapy, including the challenges of delivering the cells, promoting their retention in the body, and providing them with the time they need to integrate into tissues, Dr. Goldberg said.

    Potential advantages. Of note, with this method, “A single donor cornea can provide cells for hundreds of patients,” thus addressing donor supply limitations, Dr. Goldberg said.

    Moreover, it is less invasive than current corneal transplants—as Dr. Goldberg noted, although corneal transplant techniques have improved, the procedures are “still a significant surgery and not exactly disease modifying.” And given the relative simplicity of the process, injections could take place in the clinic, thus increasing the accessibility of this therapy for both physicians and patients.

    Testing in humans. The first human study has been completed; it involved two doses in two formulations in 21 patients with severe corneal disease. No ocular inflammation was observed, and no significant adverse effects occurred. Two patients had a mild transient increase in intraocular pressure.

    With regard to efficacy, nine of the 21 patients showed at least a 3-line gain in vision, and 14 had a significant reduction in corneal thickness.

    A multisite dose-ascending trial is slated to begin in the first quarter of 2021, Dr. Goldberg said—and one attendee responded in the chat box, “I would love to join your trial.” —Jean Shaw


    1 Xia X et al. Invest Ophthalmol Vis Sci. 2019;60(7):2438-2448.

    Watch the symposium in full. If you are registered for AAO 2020 Virtual, you have access to the archived presentations on the virtual meeting platform until Feb. 15, 2021. Log in to the virtual meeting platform: Next, from the Lobby screen, select “Sessions” from the top navigation; click “Agenda” from the drop-down menu; and search for what you are looking for.  

    Financial disclosures. Dr. Goldberg: Aerie: C; Allergan: C; Annexon: C; Astellas: S; BrightFocus: S; Carl Zeiss: C; Emerald Biosciences: C; Emmetrope Ophthalmics: O; Equinox: C; Galimedix: C; Genentech: C; GVRI: S; IrisVision: C; Mtec: S;  Nicox: C; NIH: S; Novartis: C; PH Pharma: S; Quark: C; Regeneron: C; RenetX: C; Santen: C; TwoXar: C; U.S. Department of Defense: S.

    Disclosure key. C = Consultant/Advisor; E = Employee; L = Speakers bureau; O = Equity owner; P = Patents/Royalty; S = Grant support.

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