NEI director's message

Dear Friends and Colleagues,

As many of you are aware, during the summer 2019, I stepped down from the National Eye Institute, and transitioned to a faculty position at the University of California at Davis where I have the opportunity to establish a new Center for Ocular Regenerative Therapy, CORT. Consequently, this will be my final ARVO column as NEI director.

When I became the NEI director in 2001, science was at an inflection point. Advances in vision disease research were coming to be powered by genetics and by sequencing the complete human genome. These new tools facilitated probing the function of cells and tissues at the molecular level. They have opened new windows to exploring hypothesis-driven translational studies that ultimately will ameliorate human vision disease. It has been a privilege to guide the nation’s vision research agency for nearly two decades, during a time of tremendous scientific progress.

Researchers have made rapid advances in many areas since then, in genomics, neuroscience, imaging, stem cell biology, and many other fields that impinge on the eye and visual system. I believe that both fundamental and ‘applied’ translational research are essential parts of the seamless fabric of new knowledge. Setting these in opposition in the biomedical realm is a false dichotomy. Vision researchers increasingly recognize that the more these realms interact, the more they mutually prosper.

One cannot compress all the advances of two decades into a few sentences. But it is worth commenting on a few of the seminal insights we gained as we developed new understanding of the pathobiology of diseases that steal vision from too many Americans and peoples across the globe. In 2005, NEI-supported researchers reported results from a genome-wide association study that identified the first of many key genes involved in age-related macular degeneration (AMD). Soon afterward, NEI support of fundamental and applied research led to successful human gene therapy, for Leber Congenital Amaurosis that severely limits vision from infancy, and this progressed to the first ocular gene therapy approved by the FDA, in 2017.

Possibilities for regenerative medical therapies are now gaining traction, and the first human clinical trial will soon be underway using replacement tissue deriving from patient-derived induced pluripotent stem cells and transformed into sheets of retinal pigment epithelium cells, targeting treatment for AMD from geographic atrophy. And just this year, NEI investments in neuroscience led to the first treatment for vision loss with cortical implants.

These notable firsts merely touch on the remarkable progress that the vision community has made in basic and clinical research over these two decades.

Working with the vision community, the NEI launched the Audacious Goals Initiative (AGI), to catalyze development of technologies, research models, and key datasets critical for the translation of regenerative treatments to the clinic. During the last five years, three AGI consortia have moved forward with high resolution functional imaging of the retina and to understand the retina further at the single-cell level. The program continues to provide key funding opportunities for those interested in regenerative medicine. Research supported through AGI and other programs at the NEI is poised to make it possible to restore functional vision to the blind.

Currently the NEI is enabling cutting edge-research in genetics and bioinformatics, big data and artificial intelligence, and more. Beginning with eyeGENE, our database of vision-related genetic information, and continuing with a growing list of freely available datasets, NEI is providing infrastructure and guidance on making such datasets interoperable and reusable.

As the Institute transitions into its next chapter, I am pleased that NEI Deputy Director Santa Tumminia, PhD, will serve as acting director. Dr. Tumminia has filled a number of top roles in the NEI administration since 2003, and I am confident she will ably guide the institute during the search for my successor. A national search will be conducted for the next director of NEI. This will be handled through the NIH Office of the Director, and the search likely will conclude some time in 2020.

The achievements listed here were only possible through the contributions of countless scientists, clinicians, and, of course, clinical study volunteers. Throughout this time, the ARVO organization and ARVO members have been good partners in advancing vision research. My thanks to all! Working with you during this remarkable journey of discovery has been a wonderful experience and a remarkable scientific opportunity, and I leave the institute firmly in the midst of an exciting translational era.

I look forward to seeing you in Baltimore at the 2020 ARVO Annual Meeting!

Paul A. Sieving, MD, PhD