New treatments for pediatric glaucoma: preventing long-term vision loss in pediatric glaucoma patients

Background: Pediatric glaucoma patients often follow the same course of disease that adult glaucoma patients do, with the exception that, as a primary diagnosis, pediatric glaucoma is often due to developmental structural abnormalities. This means the only option for these infants or young children is surgery which incurs higher risk the younger the child is. As such there is a significant health and economic need to develop safe medications for pediatric glaucoma patients to reduce the risk of early vision loss so that surgery can be performed at the most opportune time.

Issue: There is a pressing need to address pediatric glaucoma and to develop new diagnostic techniques to prevent long-term loss of independence and mobility.

Workplan: Specifically, this program will: (1) Assess mitochondrial and metabolic features at different time points in a well characterized pediatric glaucoma model; (2) Target NAD-metabolism to test a new neuroprotective strategy which would have rapid clinical translational potential; and (3) Further this program by developing a novel gene therapy strategy to provide a single, long-term treatment for pediatric glaucoma.

Meaning: The outcome of this research, combined with our nicotinamide trials in adult glaucoma patients, will provide the pre-clinical evidence required to start assessing nicotinamide treatment in pediatric glaucoma patients.