Wednesday, November 09, 2011

Fighting Retinal Disease: The Promise of New Approaches

Dr. Stephen Rose, the Chief Research Officer of the Foundation Fighting Blindness has just written an article about emerging treatments for retinal diseases, including the use of stem cells, gene therapy and new pharmaceutical approaches. I feel it is important for those of you on the front lines fighting these diseases and those of you who have them, to know that we are getting closer and closer to solving some of the questions about saving sight, and perhaps even bringing back lost sight.

Please take a look at Dr. Rose’s words and dig deeper into the resources available on the FFB website. (I have not included the links that were included with some of his remarks, but you can go to the original on the web and check them out for yourselves.)

What Emerging Treatment Will Work Best for Me?

Written by Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness

November 8, 2011 - Hope has never run higher in the fight against retinal degenerative diseases, thanks to clinical trials now underway for gene, stem cell and pharmaceutical therapies. As a result, people affected by these vision-robbing conditions are naturally eager to figure out what emerging treatment approach is going to work best for them.

While it is tempting for someone affected to focus on a "magic bullet" to stop or reverse their disease, I strongly encourage people to consider the Foundation's comprehensive portfolio of emerging treatments when thinking about the future of their vision. Here are three important reasons why:

We can't predict how future research will unfold

Science always has surprises. As an example, until a year or two ago, we believed that corrective gene therapy would be suitable only for treating early-stage disease when a person had a lot of photoreceptors left to save. But recently, Foundation-funded researchers from the University of California, Berkeley and other groups are using gene therapy to enable ganglion cells -- cells in the retina that survive long after photoreceptors are lost -- to provide vision. This research advance has now put gene therapy on the map for potentially reversing total blindness. Previously, we thought that only stem cells or artificial retinas could restore vision in advanced disease.

We need to have back-up plans

In the next couple of years, as we see a big increase in the number of clinical trials for inherited retinal diseases, some setbacks will be inevitable. We need to keep in mind that clinical trials are experiments and, invariably, will not always achieve optimal results. It is critical that we have multiple treatments available, or in the pipeline, for each disease, in case a promising course of action does not pan out. This is one reason pharmaceutical therapies are so important. Generally speaking, they can treat a broader range of conditions than, for example, a gene therapy directed at a specific genetic defect. I envision pharmaceuticals often serving as bridges, or as alternatives, to more targeted therapies. 

Treatment decisions will be personal

Several factors will play a role in determining which treatments might work best for an individual, including his or her genetic profile, stage of disease, age and even tolerance for risk. Let's say in the future that a middle-aged person with retinitis pigmentosa has been taking a drug for many years that's done a good job preserving vision, and along comes a new gene or stem cell therapy. Does that person try a new treatment or stick with what is known to work? There is no right or wrong course of action. What's important is that we fund a diversified portfolio of research, so patients have options. Multiple treatment alternatives may seem like a luxury now, but we are working hard to make that day a reality as soon as possible.

Find out more about Foundation research

The Foundation currently funds 130 grants at 73 institutions around the world. Grants are selected through a rigorous review process conducted by the Foundation's Scientific Advisory Board, which is comprised of the world's top retinal researchers. A complete list of the Foundation's grants is at:


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