For those of us on the front lines of fighting blindness — whether we’re raising funds, conducting  research or stand to personally benefit from the results — the device’s approval is one of the most exciting milestones in vision restoration ever achieved.

The Argus II provides only rudimentary vision; users have recognized doorways, sidewalks, flatware on a table and lights on a city skyline. But the device is enabling people who were completely blind to see. That translates into improved mobility, independence and quality of life

The Argus II’s ability to restore some basic vision is remarkable, but people want to see much more of the world — faces of loved ones, text on a computer screen, paintings in museums and so on. A device that provides the ability to perceive more detail will not only help those without any eyesight; it will also benefit many people with varying degrees of vision loss.

Developing such a device is the next challenge for companies like Second Sight. While the Argus II is equipped with a 60-electrode, or –pixel, chip implanted in the retina, the California-based company plans to develop a 256-electrode prosthesis. But it is still several years away from making it available.

There are several other companies in the artificial retina arena, with Retina Implant AG, of Germany, being the furthest along. Its device, the Alpha IMS, is a 1,500-pixel chip and, unlike the Argus II, does not require an external video camera. Approximately 36 people have used the Alpha IMS in clinical trials. Some have recognized objects, such as doorknobs and telephones. One person saw the movements of a wild goose. Another read store signs.

The company’s clinical trial is underway in Germany, Hong Kong and the United Kingdom, and it is planning to extend the study to Wills Eye Institute in Philadelphia. Retina Implant AG is working to overcome some technical and surgical challenges, and has not indicated when they expect to apply for regulatory approval in the United States or Europe.

A research group from Stanford University is developing a 5,000-pixel system known as the Photovoltaic Retinal Prosthesis, which it believes will provide more detailed vision than the alternatives. The group is hoping to launch a clinical trial within two years. With this device, images are captured by a tiny video camera mounted on a pair of glasses and converted to near-infrared (NIR) light. The NIR beam is routed to multiple one-millimeter chips implanted underneath the recipient’s retina and converted to electrical signals that are sent back to the brain through the optic nerve.

Other groups developing artificial retinas include: Bionic Vision Australia, Nano Retina in Israel, the Boston Retina Implant Project and several organizations in Japan. This is an active and innovative field, though developing this cutting-edge technology is no small feat. Stay tuned — I’ll report on the progress of these efforts as it is made.

Editor’s note: The photos above, which appeared in the spring 2013 issue of In Focus, the Foundation’s newsletter, were incorrectly described as being Second Sight’s Argus II device. They are Retina Implant AG’s Alpha IMS.

Photos provided by Retina Implant AG

Ultimately, it is as important to design a good clinical trial as it is to develop a good treatment. We could have the best treatment ever devised, but without a good human study to demonstrate its safety and efficacy, we’ll never obtain U.S. Food and Drug Administration approval, to get it to the people who need it.

Designing a clinical trial for retinal disease therapies is challenging for many reasons. First, retinal diseases often progress slowly and affect vision in ways that are not easy to measure. Often, evaluating visual acuity by having someone read an eye chart doesn’t tell us if a treatment is actually saving vision. We may need to look at the person’s peripheral vision or ability to adapt to dark settings.

Observing structural changes in the retina itself may enable us to more quickly determine if a therapy is benefiting vision. Figuring out the best “outcome measures,” as we call them, for use in a clinical trial is one of the most important goals of ProgSTAR.

ProgSTAR will also help identify the best potential participants for a clinical trial of a Stargardt disease therapy. Will people with early-stage or late-stage disease be more likely to respond to a therapy?  How long will we need to monitor them to observe a response? We need answers to these questions to implement treatment studies that are cost-effective and expedient.

Not only do we need the right patients for a clinical trial; we need good clinical researchers. The Foundation has assembled the world’s best Stargardt disease experts for ProgSTAR. Led by Dr. Hendrik Scholl, of the Wilmer Eye Institute at Johns Hopkins, these investigators are unmatched in their understanding of Stargardt disease, and participation in the study will advance their knowledge even further. Dr. Patricia Zilliox, the Foundation’s chief drug development officer, serves as project director. She is well-qualified, with more than 30 years of industry experience.

ProgSTAR is monitoring approximately 200 people enrolled at nine clinical centers. They will be monitored for two years, but researchers are also looking at retrospective data on patients to get a better picture of how the disease and vision loss progress. ProgSTAR is a “by invitation-only” party; investigators are recruiting only their existing patients to participate.

It is rare for a non-profit foundation to fund a natural history study, because they are expensive — ProgSTAR will cost at least $3 million — and they don’t provide an immediate return of a new therapy. But, as I’ve just explained, these studies are invaluable for moving treatments forward for inherited retinal diseases; they bring us a big step closer to a cure.

Pictured, above: Dr. Hendrik Scholl conducts an electroretinogram, or ERG, with a patient at the Wilmer Eye Institute.

What’s signficant about many emerging neuroprotective therapies for the retina is that they have the potential to treat a wide range of diseases, regardless of the genetic mutation causing vision loss. With about 200 genes linked to retinal diseases, this is a huge plus. They might also be used in conjunction with a genetic therapy to enhance its vision-saving effects.

Though neuroprotection usually isn’t addressing the root cause of the disease, it may be nearly as beneficial as a permanent fix, if useful vision can be saved for a person’s lifetime.

Neuroprotective treatments are being developed in many forms — proteins, drugs (small molecules) and nutrients. And there are several ways to get a neuroprotective therapy to the retina, including eye drops and oral medications.

Imagine having a therapy factory in the retina — in the forms of transplanted cells or genes — that provides continual release of neuroprotective proteins. Well, researchers are working on those, too.

Here are three examples of emerging neuroprotective therapies for the retina:

Protecting Mitochondria — MitoChem Therapeutics, a Foundation-funded  start-up company, has identified two compounds, which show potential for saving vision for people affected by several retinal diseases, including retinitis pigmentosa (RP), cone-rod dystrophy, Bardet-Biedl syndrome, Usher syndrome, Stargardt disease and age-related macular degeneration (AMD).

The compounds work by protecting mitochondria, the power supplies for all cells in the body, including those in the retina. Investigators have refined the compounds to the point where they save virtually all of the photoreceptors in a mouse model of retinal degeneration. They’ve also demonstrated that eye drops can effectively deliver large amounts of one compound to retinas in eyes comparable in size to those in humans. Eye drops are beneficial, because they minimize potential systemic side effects.

Docosahexaenoic Acid (DHA) — DHA is an important structural component of cells in the brain and the retina. It appears to have many neuroprotective properties and is prescribed for a variety of conditions, including heart disease. Perhaps most relevant to retinal degenerative diseases, DHA can reduce the destructive effects of inflammation and oxidative stress. It also helps maintain the structure and metabolism of photoreceptors.

The Retina Foundation of the Southwest is completing a Foundation-funded clinical study of DHA for males with X-linked RP. Previous clinical studies conducted by Dr. Eliot Berson show that vitamin A combined with DHA can slow vision loss in people with RP. In 2014, the National Eye Institute will complete a clinical study of DHA and other nutrients for people at risk of advanced AMD. DHA can be obtained by eating salmon, tuna and other coldwater fish, or by taking fish-oil or algae supplements.

Rod-Derived Cone Viability Factor (RdCVF) — Drs. José Sahel and Thierry Léveillard, of the Institut de la Vision in Paris, received the Foundation’s Trustee Award for their discovery of Rod-derived Cone Viability Factor (RdCVF), a protein that preserves and rescues cones, the cells in the retina that provide central and color vision. They are now developing a gene therapy that provides sustained delivery of RdCVF after a single treatment. The protein could be beneficial to people affected by a broad range of retinal diseases, including several forms of retinitis pigmentosa. With Foundation funding, the researchers are working to move their RdCVF gene therapy into a clinical trial.

There are several other emerging neuroprotective therapies in the Foundation’s research portfolio. Stay tuned to Eye on the Cure and the Foundation’s website for updates on these emerging cross-cutting treatments.

Pictured, above: Some neuroprotective therapies may be delivered by eye drops. (Photo courtesy of the National Eye Institute.)

While essential to fighting blindness and many other conditions, translational research is painstaking and very expensive. It comes with risks and requires extensive clinical and regulatory expertise. It is no cake-walk.

But as the following video shows, Foundation-funded researchers developing a variety of treatments — including stem-cell, genetic and pharmaceutical therapies — are succeeding in meeting these challenges. Their determination and confidence are quite inspiring. Check out the video — I think you’ll agree.

Market approval of the Argus II is especially poignant because FFB funded an early stage of its development, and we’ve closely followed and reported on its development ever since. For these reasons, Foundation staff and board members were quoted, last week, in articles on the device in The New York Times and The Washington Post. Our offices were also paid a visit by Al Jazeera TV, which aired this piece over the weekend.

But the buzz extends even farther, with articles and posts showing up in numerous blogs and publications, including USA Today, Forbes, Popular Science, Bloomberg, The Wall Street Journal and the Los Angeles Times.

We congratulate Second Sight and are honored to have played a role in the Argus II’s development. And, as always, we look forward to reporting more good news about treatments and technological advances for restoring vision for people with  retinal diseases in the future.

Pictured, above: the Argus II implanted on the retina