I am happy to report that the panel did, in fact, vote to recommend marketing approval, and I think the heartfelt testimonies of those who used the Argus II in clinical trials helped move the panel toward that decision. The panel’s recommendation bodes well for final FDA approval, which will hopefully arrive in the not-too-distant future.

While Dean testified pragmatically — no surprise, he is a lawyer, after all — others who spoke about their experiences with the Argus II were quite emotional. For example, Cathy Blake, also blind from RP, became tearful as she explained how one of the most important benefits of using the Argus II is the ability to see movement.

“What I really love most about the device is I can tell if people walk by me or there are cars moving in the street,” she said. “I was even able to see fireworks — the flashes falling.”

David Brint, a member of the Foundation’s Board of Directors and Science Liaison Committee, spoke compellingly about his son, Alan, a 15-year-old who has very little vision due to Leber congenital amaurosis. David said, “My son can see where there are trees or a sidewalk. He sometimes sees our dogs. But he asked me if he will someday ‘go dark.’ He said he’d be happy if he could just keep what little vision he had. The Argus II might be a way to restore that vision should it be lost later in life.”

Video of the Argus II Retinal Prosthesis, courtesy of Second Sight

Most of the 10-hour hearing focused on safety and efficacy data from the clinical trials. In the morning, both the FDA and Second Sight, developer of the Argus II, presented data. After the patient testimony in the afternoon, the FDA panel had several questions for Second Sight representatives and then deliberated openly among themselves.

FDA approval of the Argus II would be truly historic and groundbreaking. While several clinical trials for potential sight-saving treatments are underway, and Dr. Eliot Berson’s vitamin A regimen for slowing RP is available, there are currently no FDA-approved therapies for rare inherited retinal degenerations.

FDA approval of the device would also be a big boost for Second Sight, which has received marketing approval for the device in Europe and has plans to enhance the Argus II to provide even better vision than it does today.

Right now, the future is looking bright for the Argus II, and I’ll be the first to let you know about the latest developments in the quest to bring it to market.

The alternatives are: Lucentis, a monthly treatment approved by the FDA in 2006; Avastin, an equally effective, low-cost, off-label alternative to Lucentis; and Eylea, which was approved by the FDA in 2011, and is as effective as Lucentis, but with fewer treatments.

These therapies are having a dramatic impact on vision around the world. For example, Danish researchers reported earlier this year that the rate of legal blindness from wet AMD in their country decreased by 50 percent from the year 2000 to 2010, with most of the reduction occurring after 2006, when Lucentis and Avastin became widely used.

That’s the good news.

The not-so-good news is that, even though these treatments work, wet AMD invariably returns in all patients, usually within a few months after the treatment is stopped, and retreatment (i.e., additional ocular injections) is necessary. Some retinal doctors continue monthly treatment for the life of the patient even after the leaky blood vessels that cause vision loss from wet AMD are no longer apparent. And as you might imagine, or know firsthand, ocular injections are inconvenient, can cause some discomfort and carry small risks.

However, there are potentially better options on the horizon. Three companies — Oxford BioMedica, Genzyme, and Avalanche — are conducting early-stage clinical trials of gene therapies that could potentially halt wet AMD for several years, perhaps a lifetime, through a one-time ocular injection. These treatments work by delivering copies of a gene to the retina that operate like little biotech factories, providing continual release of a protein that prevents the growth of vision-robbing leaky blood vessels. While these gene therapies are in safety studies for now — we won’t know if they prevent vision loss for perhaps a few years — we’re excited about their potential.

What is also exciting for me is that the technologies used to deliver the therapeutic gene to the retina — manmade viruses — are very similar to those developed earlier by Foundation-funded researchers for the treatment of rare diseases like Leber congenital amaurosis (LCA), retinitis pigmentosa, Usher syndrome, choroideremia, and Stargardt disease.

One of the big challenges in treating rare inherited retinal degenerations is that there are so many of them — several dozen, in fact. But, fortunately, many of the treatment approaches we fund apply to many diseases. So, while a therapy for a rare disease like LCA might initially target a few hundred or a few thousand people, it might ultimately help millions. When it comes to fighting blindness, we are all in it together.

Patricia Zilliox, Ph.D., the Foundation’s chief drug development officer

Of course, it would be nice if we could test a potential treatment in just four or five mice and, if it were to save vision, bypass the regulatory process and immediately get it out to everyone who needs it. But by being that hasty in the real world, we wouldn’t fight blindness at all; instead, we might make people sick, impair their vision even more, and put their lives at risk.

Therein lies the indispensable regulatory role of the FDA, its European counterpart, the European Medicines Agency (EMA), and other agencies around the world — to protect consumers by ensuring, as best as possible, that a treatment is safe and beneficial.

As the Foundation’s chief drug development officer, I have to take the FDA into consideration, directly or indirectly, in just about every decision I make. And, having worked for three decades with the FDA and the EMA, I’ve developed an understanding of what it takes to get authorization for clinical trials and marketing approvals for treatments that perform well in those studies.

So what is the FDA looking for? First and foremost, it wants to ensure that a potential treatment is safe. That means extensive testing in the lab and throughout human studies to demonstrate that the therapy is not toxic and not causing adverse events in the patients. Safety and toxicology studies must be performed and documented meticulously.

An oral drug has the potential to affect virtually every part of the body, so related safety studies must be very comprehensive. Does the drug affect the cardiac system? Is it causing an immune reaction? Does the treatment increase risk of cancer or birth defects for pregnant women? Does it lead to mood changes or mental health issues? All of these potential effects must be watched for in both lab studies and clinical trials.

Demonstrating to the FDA that a drug is saving or restoring vision is also challenging and expensive, namely because retinal diseases vary so much in their rate of progression and how they impact vision. As a result, evaluating efficacy usually requires large numbers of patients, several types of vision tests and sophisticated imaging of the retina. A recent article on the Foundation’s website describes many of the tests and imaging techniques used in clinical trials to evaluate changes in vision.

Finally, a company or institution that brings a treatment to the market must demonstrate that it is following the FDA’s Good Manufacturing Practices (GMP) to make certain that each pill, eye drop or injection is produced in a sterile environment and that there is no variation in doses, packaging and shipments. This requires specialized institutional oversight and expertise as well as rigorous production standards.

Once a treatment is FDA-approved, the company or institution distributing the drug is obligated to continue to collect and report information on side effects and adverse events. Post-marketing surveillance can reveal problems or limitations with a treatment that were not identified in clinical trials.

Ultimately, when anyone goes to a pharmacy to pick up a prescription or visit a doctor for a treatment, the FDA has overseen the development and production of those therapies at virtually every step to ensure they are safe and effective. And, as the Foundation moves treatments through clinical trials and out to the market, we and our partners are working hard to follow FDA regulations and standards, so we can get approval to deliver them to the people with retinal degenerative diseases who need them.

Patricia Zilliox, Ph.D., is the Foundation’s chief drug development officer. She oversees the planning and execution of clinical trials through the Foundation’s National Eye and Evaluation Research Network, and partners with institutions and companies around the world to advance promising treatments to patients affected with blinding retinal diseases.