Q: What prompted the creation of Foundation Fighting Blindness?
The Foundation Fighting Blindness (FFB) was created in 1971 by a passionate group of people who were impacted by vision-robbing retinal diseases, including Lulie and Gordon Gund, who serves as chairman today. Little was known about these degenerative eye conditions at the time, and almost no research was being done to better understand and treat them. Experiencing similar frustrations and challenges, the founders realized it was up to them to jumpstart this field of research, and they shared the same determination to find answers. While they knew little about how or where to start building momentum toward treatments and cures, those pioneering families sensed that no one else would do it for them.
They established the Berman-Gund Laboratory at the Massachusetts Eye and Ear Infirmary at Harvard University, which was the foundation's first research project. This lab is still doing innovative work today, along with 13 other foundation-funded centers focused on retinal research, and a cadre of scientists receiving grants around the world.
Back in 1971, there was essentially no hope. People newly diagnosed with a retinal disease were often told to go home and learn Braille because there was nothing that could be done. Today, that message is hopeful. Thanks in large part to the Foundation Fighting Blindness, reversing vision loss is a reality, and clinical trials are now restoring sight in patients. We're at a critical point with the pieces in place to help millions, and securing the necessary funding to continue this momentum will get us to our end goal.
Q: What were the challenges in retinal disease research that it was felt FFB could help address?
Because most retinal diseases—like retinitis pigmentosa, Usher syndrome, and Stargardt disease— are rare and affect small populations, commercial interest in developing treatments is limited. So, the founders and our initial supporters learned early on that the foundation would need to be largely responsible for shepherding promising sight-saving approaches from bench to bedside.
Another challenge has been the complex nature of retinal diseases because they stem from hundreds of possible genetic mutations and affect people's vision differently at varying rates of degeneration. So, a significant amount of research energy, especially earlier on, focused on understanding the genetics of these conditions and how they develop. This basic research continues today, but because of more than four decades of scientific progress, there are now at least 15 clinical trials underway for retinal disease therapies.
Over the years, the Foundation Fighting Blindness has also addressed the need to fill the pipeline of bright talent committed to retinal research. Through our Alan Laties Career Development program, ambitious young clinician-scientists receive research grants, with the hope that they will continue on the career path of sight-saving retinal research and patient care. Supporting these up-and-coming innovators is an investment in future intellectual resources and key to fueling the momentum we have going.
Q: Can you tell us a bit about FFB's research strategies and how they help overcome the barriers you've identified?
With sequestration and budget challenges negatively impacting the National Eye Institute, the foundation's role in advancing treatments is more important than ever. By investing in cutting-edge approaches early on that our Scientific Advisory Board deems promising—when others won't take on the risk—we are filling a key void in the process. The function of our venture philanthropy is to shepherd potential vision-saving therapies through necessary lab research and into clinical trials—through the so-called "valley of death"—to the point where venture capitalists or pharmaceutical companies want to invest at a high level.
We can be viewed as a "matchmaker of science," bringing together researchers on the front lines, small biotechs, big pharma, and life sciences venture capitalists to foster collaboration and help attract the major funding necessary to bring treatments to the marketplace. To boost our focus on translational research, we created the Foundation Fighting Blindness Clinical Research Institute, a support organization that is developing a bridge between scientific, clinical, governmental, pharmaceutical and financial communities to advance treatments into and through clinical trials.
And, our Translational Research Acceleration Program (TRAP) supports some of the world's leading retinal researchers working toward launching new clinical trials within the next few years. Their research spans the areas of genetics, drug discovery, stem cell treatments, and gene therapy, demonstrating our pursuit of multiple research approaches. Since there likely isn't a "one size fits all" treatment for the spectrum of retinal diseases, exploring different cross-cutting methods is another core strategy.
Q: What do you consider FFB's greatest accomplishments to date?
Raising more than half a billion dollars toward our mission since the foundation's inception in 1971 has gotten us to the point of actually restoring sight. In landmark, ongoing clinical trials of a novel gene therapy, which were made possible by decades of foundation funding, dozens of children and adults have had significant vision restored, including a boy who was able to put away his white cane and play baseball after the treatment. This medical breakthrough has led to additional gene therapy human studies for a variety of retinal diseases.
Through our Clinical Research Institute, the foundation has launched its first-ever clinical trial, evaluating a drug called valproic acid in slowing vision loss in people with autosomal dominant retinitis pigmentosa. Nearly every emerging retinal disease treatment in development has the foundation's mark, including early funding for the Argus II retinal prosthesis, which was recently the first device of its kind to receive FDA approval. We also supported much of the lab research that enabled stem cell-based clinical trials now underway for Stargardt disease and age-related macular degeneration.
Another huge accomplishment has been the development of nearly 50 Foundation Fighting Blindness chaptersthroughout the country that provide support, information, and resources to families living with retinal diseases. These networks allow affected indiviuals to connect with others and work together to raise awareness and funds in their community, through our signature events like VisionWalk and Dining in the Dark.
Q: What are your top research goals over the next year, and what will it take to reach those goals?
Because of the immense costs associated with translational research and clinical trials—tens of millions of dollars for each human study—we need to significantly ramp up our fundraising efforts to achieve our goal to launch several clinical trials over the next few years.
In the vein of venture philanthropy, we'll also continue to leverage relationships with start-up biotech companies to foster treatments. For example Applied Genetic Technologies Corporation and GenSight Biologics are companies that each received tens of millions in venture capital funding for their gene therapies, thanks to the foundation's translational research support. The foundation is also supporting a new startup company, MitoChem Therapeutics, with a $2 million investment to move its potential treatment into a clinical trial.
Within our scope of gene-based, cell-based, and pharmaceutical treatments, there are innovative new approaches we're supporting that show great promise for restoring vision. For instance, optogenetics is an up-and-coming field that has potential to help people with advanced sight loss. And, through a technology known as induced pluripotent stem cells, researchers can take blood or skin from a patient, turn the clock back on those cells so they become stem cells, and then coax them into becoming retinal cells for use as a customized treatment.
At the May annual meeting of the Association for Research in Vision and Ophthalmology, we observed that the foundation is funding about 60 percent of the retinal projects presented there, a true testament to our involvement in every aspect of this field.