Lions’ efforts in 2007 result in new therapy in 2018

Physician clinicians and researchers in the Department of Ophthalmology and Visual Sciences at the University of Iowa continue to make important research breakthroughs in the fight to combat Leber’s Congenital Amaurosis (LCA), a genetically inherited eye disease that causes babies to be born blind or children to go blind before reaching school age.

The Iowa Lions have played an important role in making this happen.

In 2007, researchers at the University joined with the Lions Clubs of Iowa to create Project 3000, the goal of which was to find people born blind or adults who became blind as children, test them, and find the roughly 3,000 people in the U.S. with LCA. The researchers worked with other researchers across the country, who also enlisted their local Lions Clubs in the efforts. The Iowa Lions canvassed their own club communities to locate individuals throughout Iowa who might have LCA, and offer them genetic testing.

The Iowa Lions responded with great enthusiasm and identified more people in Iowa with LCA than would have been expected given its population.

Since that time, researchers have been working toward a gene therapy to help treat patients with LCA, and in 2017 their efforts resulted in helping to restore sight and gaining FDA approval for the treatment.

In the study, 29 patients between the ages of four and 44 were given the same gene therapy, called SPK-RPE65 and developed by Spark Therapeutics, Inc. Prior to the therapy, none of the patients could see well enough to walk unassisted in a regularly lit room. They were tested on their ability to navigate a room set up as a kind of obstacle course.

The treatment is injected directly into the eye. In the final stage of the trial, patients were directed by arrows through a mobility course in seven different light levels. The course changed with each change of light level. The lowest level of light was that of a moonless summer night, and the brightest was that of a well-lit office.

After a year, patients treated in both eyes improved by 1.9 light levels, and their visual acuity improved by eight letters, roughly one and a half lines on an eye chart.

In October 2017, an FDA advisory panel voted unanimously to recommend approval of this treatment. It is expected to receive approval in January 2018 and become the first gene therapy in the U.S. for a genetically inherited disease, and the first in which the patient directly receives a corrective gene. This news generated significant national media coverage on CBS, NPR, CNN, and NBC, and other media outlets.

And while this breakthrough has obvious immediate benefits from those who suffer from LCA, this treatment may also open the door for future gene therapy treatments for other disorders. We are proud to be a Lions eye bank, and proud of the efforts of the Lions Clubs of Iowa to invest in beginning research efforts. It was an 11-year journey from locating patients who might have the disease to a clinical trial and FDA approval for a treatment to restore sight, and the results could not have been achieved in such a timely manner without the efforts of the Lions Clubs of Iowa in 2007.

The Iowa Lions have a legacy of supporting cutting edge vision research, and recently have stepped up yet again. The Iowa Lions Foundation recently voted to award $29,000 of the $65,000 it is raising for Iowa Lions Eye Bank research team to buy a much-needed piece of research equipment, the Chemidoc MP Imaging System. This is critical for our research into diabetes, which is rising in the population and can present complications for corneal transplants, both for diabetic donors and for diabetic recipients.

We are focused on finding therapies to increase the success rate of diabetic-related transplants. The Chemidoc MP Imaging System will allow us to observe how proteins behave in corneal tissue. Keeping these proteins under control can prevent transplant failure or disease in diabetic corneal tissue, preserving vision for a longer period of time. Discoveries like these have an enormous impact on the quality of life for the more than 45,000 cornea transplant recipients each year.

Without the support of the Iowa Lions, breakthroughs like the recent LCA gene therapy treatment and those we hope to achieve in our diabetes research would be much more difficult to achieve. We at the Iowa Lions Eye Bank and the University of Iowa Department of Ophthalmology and Visual Sciences thank the Iowa Lions for their tireless contributions to the preservation and restoration of sight.