We Orioles fans appreciate that Adam isn’t marveling at how well his eyes are tracking the ball during its quick, 300-foot journey. That, of course, might distract him from catching it.

But for many researchers fighting blindness, understanding the complex process of vision, and how the retina makes it possible, is their game.

Take, for example, Erika Ellis, a medical student at the University of California, San Diego, and Howard Hughes Medical Institute research fellow, who is receiving a one-year career development award from FFB to study retinal ganglion cells. Erika will be researching how these cells refine and package visual information and send it through the optic nerve to the brain, where the final images are created and interpreted.

While the process of seeing begins when photoreceptors convert light into electrical signals, it’s up to many other types of downstream retinal cells — including ganglion, amacrine and bipolar cells — to contextualize and enhance the signals so we can perceive motion, contrast, edges and boundaries and other visual details.

Researchers like Erika are particularly interested in how ganglion cells map to different regions of the brain. There are approximately one million axons — fibers in the optic nerve — connecting the retina’s ganglion cells to the brain, so the task is daunting. But documenting the brain-retina relationship will enable experts to better understand how they work together and how well emerging retinal treatments are restoring vision.

Ganglion cells are also an attractive target for vision-restoring treatments, because they survive long after photoreceptors degenerate from diseases like retinitis pigmentosa and macular degeneration. Emerging optogenetic therapies are designed to empower ganglion cells to respond to light, so they can function somewhat like photoreceptors and restore vision. While their research is at an early stage, it holds promise for people who have lost their photoreceptors to the most advanced retinal conditions.

If you are interested in learning more about ganglion cells, optogenetics and the Foundation’s diverse research portfolio, there’s still time to register for our VISIONS 2013 conference, taking place in Baltimore June 27-30. You’ll also get the opportunity to meet nearly 50  of the Foundation’s research all–stars.

And if you happen to be a baseball fan, the Yankees are also in town that weekend, playing at Camden Yards, right down the road from the conference hotel. Come root for Oriole standouts like Adam, Chris Davis, Manny Machado, Matt Wieters and Nick Markakis. The Yankees have some players as well, but I can’t recall who they are.

Pictured, top, Baltimore Orioles centerfielder, and Golden Glove recipient, Adam Jones; and, above, HHMI-FFB Medical Fellow Erika Ellis.

Want to know about gene therapy? Our playlist features four videos, one each from four renowned researchers, including Dr. Eric Pierce, chairman of FFB’s Scientific Advisory Board, who does a great job summing up the vital role research plays in finding treatments and cures:

We also offer playlists covering stem cell therapy and gene discovery. And you won’t want to miss the video on why making donations to FFB’s research is so important at this crucial time:

To view all of the Foundation’s playlists, click here.

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.

In the Proceedings of the National Academy of Sciences, the investigators report sustained vision improvement of three years, thus far, in the treated eyes of participants. However, they also note that retinal degeneration, the loss of photoreceptors, continued in the treated eyes at a rate that was similar to untreated eyes.

Before I elaborate on the questions these results raise, it is important to be clear on what we do know from the LCA gene therapy human studies underway.

First, the Pennsylvania-Florida team has treated 15 patients, providing vision improvements without any serious adverse events. Establishing safety is the primary goal of this Phase I/II study, and, thus far, the team has done that.

Second, patients in the other gene therapy clinical trials for LCA, including the study at the Children’s Hospital of Philadelphia (CHOP), have also experienced vision improvements without serious adverse events. CHOP has also reported good results in the treatment of some patients’ second eyes. More than 40 people have been treated in six LCA gene therapy clinical trials.

The most important and immediate question is: Are the other LCA (RPE65) gene therapy clinical trials also observing loss of photoreceptors in the treated eyes of their patients? Their teams haven’t formally reported information on rates of degeneration, but I’m currently investigating this to learn what they do know at this juncture. If they aren’t seeing loss of photoreceptors, then we need to figure out why there are differences in retinal degeneration in treated eyes among the different studies.

If investigators at the other trials are observing continued retinal degeneration, it may mean that we need a more advanced form of gene therapy, to ensure that all of a patient’s photoreceptors are treated. Such a therapy might do a better job of slowing or stopping degeneration and preserving vision over a longer period of time.

Or, we may have to combine gene therapy with a drug or supplement that can stave off degeneration. These recent study results may also underscore the benefit of treating a patient early, before the degenerative process has time to gain momentum and becomes more difficult to stop.

I am very interested to see what will be observed in the early-stage gene therapy clinical trials underway for other retinal diseases. Thus far, we only have preliminary safety reports, so we’ll need to wait and see how these therapies impact both vision and retinal degeneration.

One of the big challenges in fighting blindness is the diversity of retinal degenerative diseases, and the likelihood that one approach to gene therapy will not work for all conditions. But the good news is that the retinal research community isn’t putting all of its eggs in one gene therapy basket.

As current gene therapy clinical trials move forward, and more are launched in the next two to three years, we’re going to learn a lot more about which approaches work well and which need refinement. I look forward to reporting additional information on these gene therapy studies as I get it.