Congressional Briefing Underscores the Importance of Vision Research Funding

The annual cost of vision loss in America is $68 billion, because of expenses related to health care and loss of independence. Furthermore, conditions such as age-related macular degeneration - the leading cause of blindness in Americans 55 and older - greatly impact quality of life, leaving people unable to drive, read, or recognize the faces of loved ones.

"Today the message is very clear: There is much that can and must be done to overcome these blinding conditions," said Congressman Pete Sessions (R-TX), who spoke at the briefing. "It is our responsibility through good public policy to recognize that we have the ability to find cures for these diseases."

Congressman Sessions, who has a teenage son affected by a retinal condition known as retinitis pigmentosa (RP), added, "When you have a 16-year old son who wants to be a physician, and has a great life ahead of him, you're crushed when you learn he is losing his eyesight to RP."

The Congressman noted that it is in the public's best interest to ensure that the best, most-promising research technologies are investigated and employed. He emphasized the great potential of stem cell therapies for treating vision loss. "We need to find out what stem cells can do for us in order to make wise public policy," he said.

Stephen Rose, Ph.D., Chief Research Officer of the Foundation Fighting Blindness (FFB), and Paul Sieving, M.D., Ph.D., Director of the National Eye Institute (NEI), discussed why partnerships between government and the private sector are beneficial to advancing research. Both Dr. Rose and Dr. Sieving noted that an FFB-NEI partnership helped to advance a treatment called Encapsulated Cell Technology (ECT), which is now in Phase II/III studies across the country for treatment of a variety of retinal degenerative conditions including retinitis pigmentosa, dry age-related macular degeneration, and Usher syndrome (combined blindness and deafness).

"FFB infused money into Neurotech when it was a company about to close their doors, because investors lost interest in the ECT. In an FFB-funded lab study, the ECT showed promise for saving vision, so the NEI subsequently conducted Phase I safety studies of it," said Dr. Rose. During the Phase I trial, the ECT partially restored sight in some patients. More than 150 people are now enrolled in the Phase II/III studies, which are funded in part by FFB.

Dr. Sieving, who showed the tiny ECT to briefing participants, added, "What is learned in treating the eye may translate into benefits for treating other neurodegenerative diseases such as ALS, Parkinson's disease, and Alzheimer's disease. One of the advantages of working in the eye, is that it is easier to get the device into the eye than into the skull."

An FFB-NEI partnership has also been instrumental in advancing gene therapy that may cure an inherited retinal disease called Leber congenital amaurosis (LCA) - a particularly devastating disease that causes children to be born blind or with severe vision loss. Dr. Rose said, "In 1993, an NEI researcher discovered RPE65 - a gene that causes LCA. In 2000, FFB and NEI researchers used gene therapy to give vision to more than 50 dogs born with LCA caused by that gene. Now, three FFB-funded human trials of RPE65 gene therapy for LCA are underway - at Children's Hospital of Philadelphia, University of Pennsylvania, and Moorfields Eye Hospital in the U.K."

One of the most exciting reports to come out of the briefing was on a "bionic" retina that is currently in Phase II clinical studies across the country. Dr. Rose said that this artificial retina - developed by the company Second Sight - is giving people who were previously blind the ability to see shapes, light and movement. Using the device, people are now able to navigate without assistance.

Though many retinal diseases are considered to be rare or "orphan" - meaning that they affect less than 200,000 people - research for orphan diseases is a critical public health issue. Collectively, 25 million Americans are affected by some orphan disease. Dr. Sieving said that over time, many common conditions will be subdivided into smaller, genetically related groups. "Heart disease, diabetes, and cancer are all genetically driven diseases. We are finding that all common diseases in fact splinter into groups of rare genetic diseases, and we are beginning to treat these conditions based on their genetic profile."

Dr. Rose remarked that it is an extraordinarily promising time in vision research, because of the numerous human studies emerging and underway to save and restore vision. He said, "Ten years ago, a person with a retinal degenerative disease was told: There's nothing we can do for you, go home and learn Braille, you will go blind. Today, thanks to funding from the NEI and FFB, there are clinical trials underway to eradicate these diseases. There is a lot of hope."

About the National Eye Institute
The National Eye Institute (NEI) was established by Congress in 1968 to protect and prolong the vision of the American people. As one of the Federal government's National Institutes of Health (NIH), the NEI conducts and supports research that helps prevent and treat eye diseases and other disorders of vision. This research leads to sight-saving treatments, reduces visual impairment and blindness, and improves the quality of life for people of all ages. NEI-supported research has advanced our knowledge of how the visual system functions in health and disease.

About The Alliance for Eye and Vision Research
The Alliance for Eye and Vision Research (AEVR) is a 501(c)3 non-profit foundation dedicated to education about the importance of federal funding for eye and vision research.

Stephen Rose, Ph.D., Chief Research Officer of the Foundation Fighting Blindness (FFB), and Paul Sieving, M.D., Ph.D., Director of the National Eye Institute (NEI)

Congressman Sessions (R-TX) and Stephen Rose, Ph.D., Chief Research Officer of the Foundation Fighting Blindness

Stephen Rose, Ph.D., Chief Research Officer of the Foundation Fighting Blindness speaks to the attendees

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DISCLAIMER: Physicians differ in their approach to incorporating research results into their clinical practice. You should always consult with and be guided by your Physician's advice when considering treatment based on research results.