Neuroregenerative Strategies in Glaucoma

Jeffrey Goldberg, MD, PhD Stanford University


For glaucoma, the goal is to protect sight by preserving the function of retinal ganglion cells (RGCs), which make up the optic nerve. In many forms of glaucoma, damage associated with increased intraocular pressure (IOP) begins to damage the optic nerve, ultimately killing the RGCs, with subsequent associated vision loss. Dr. Goldberg is conducting a phase 2 clinical trial where he will implant into the eye a tiny device, called NT-501 encapsulated cell therapy (NT-501 ECT).  The NT-501 ECT contains cells designed to deliver a steady stream of a growth factor, called ciliary neurotrophic factor (CNTF), to test whether it can protect damage to the optic nerve, and, possibly, enhance visual function in patients with glaucoma.

In parallel, we have advanced the field of stem cell-to-retinal ganglion cell differentiation, discovering a new pathway regulating the generation of RGCs from human stem cells, and discovering that human stem cells help support the survival of RGCs after transplant.

Project Details

NCT02862938 is the identifying number for this two year phase 2 clinical trial, with the goal to enroll 60 participants, age 18 or older, with clinically stable glaucoma in one or both eyes. Participants will be randomly assigned to either a treatment group (“subjects”) or a non-treatment group (“controls”).

Subjects will have the NT-501 ECT investigational product implanted in one eye, and will be followed for 24 months. Controls will not receive an implant but will be followed in a similar manner. At a later point, controls are expected to have the option to receive the NT-501 ECT treatment.

Patients will be evaluated quarterly over 2 years. The main outcome measure being used to judge the treatment’s effectiveness is change in visual field (side/peripheral vision) at 6 months, although additional outcomes will be evaluated over the course of the two-year trial. Some of these secondary outcome measurements look at retinal nerve fiber layer thickness, contrast sensitivity (difference in luminance or color that makes an object distinguishable), best corrected visual acuity (the clarity or sharpness of vision), visual field pattern standard deviation, and structural changes in the optic nerve head (the point of exit for RGCs leaving the eye).

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