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Optic Neuropathy Program

Vision loss from optic nerve injury due to trauma, multiple sclerosis or vascular disorders such as NAION is complex and multifaceted. Successful treatment of this devastating condition is limited by three major challenges: loss of neurons, production of an impenetrable glial scar and loss of key supporting cells called oligodendrocytes. Stem cells present in the optic nerve of many animals overcome these problems to carry out repair and regeneration but in mammals like mice or humans these CNS stem cells are inhibited and remain dormant after injury. Injecting stem cells into damaged spinal nerve tracts that closely resemble the optic nerve improves function in mice and some groups have injected stem cells to treat spinal cord injury in man. These studies raise hope that stem cell replacement therapy can be employed to treat damaged optic nerve.

Beyond replacement therapy, a unique approach taken at the Neural Stem Cell Institute (NSCI) is to stimulate the endogenous stem cells naturally present but dormant within the optic nerve. Activating such endogenous stem cells by implantation of biodegradable beads that release stem cell activating growth factor addresses all three problems limiting treatment and dramatically improves function in mice with injured spinal nerve tracts that mirror optic nerve injury. The discovery of Stembeads offers a novel bioengineered method to activate endogenous stem and progenitor cells, reawakening them from dormancy and instructing them to promote repair and regeneration. NSCI is poised to accomplish the defined and critical next step treating the optic nerve directly to translate the Steambead discovery into beneficial therapy.