Ophthalmology is a promising specialty for implementing Precision Medicine because of the eye’s amenability to intervention and the significant human and economic burdens it incurs. Retinal degenerations affect 10 million Americans and account for a significant portion of the USA’s annual $51 billion dollar healthcare expenditure on ophthalmology. The prevalence of blindness is expected to double by 2020 due to the aging population, which will only exacerbate health care costs.
Ophthalmic Precision Medicine is facilitated by the eye’s relative immune privilege and accessibility, and the effects of treatment can be precisely monitored non-invasively at the resolution of a single cell with adaptive optics imaging. As a pair organ, the eye provides the ideal treatment-control conditions and distinguishes itself as the ideal system for Precision Medicine due to the low risk of rejection of gene and stem cell therapies. In fact, therapies involving embryonic stem cell transplants for macular degenerations are the only FDA-approved regenerative medicine trials currently available in the US.
At Columbia University Medical Center, the Edward S. Harkness Eye Institute's clinic has over 800 genotyped retinitis pigmentosa and juvenile macular degeneration patients awaiting FDA approval of gene therapy approaches to restore their ability to conduct activities of daily living.