At Two Blind Brothers we donate 100% of our profits to researchers and the Foundation Fighting Blindness (FFB) to find cures to blindness, and today we just got one step closer to our goal. In a World Sight Day miracle, a U.S. Food and Drug Administration (FDA) advisory panel met to vote on whether or not to recommend Luxturna, a gene therapy developed by Spark Therapeutics that could restore vision for people with Leber’s congenital amaurosis (LCA), to the FDA for approval. It’s expected that the panel will give this treatment the greenlight.
SO, WAIT…WHAT IS LUXTURNA AND HOW DOES IT WORK?
Luxturna is the trademarked name for Spark Therapeutics’ “Voretigene Neparvovec” gene therapy. Don’t worry if you’ve never heard of that term before, neither had we.
It’s just their technical term for the gene therapy that corrects the inherited mutated LCA-causing gene, i.e. the bad version. How? By injecting a copy of the unmutated, or normal version, of the gene directly into the retina.
Ouch! That sounds painful! Don’t worry Luxturna is meant to be a one-time treatment and as they say, no pain, no gain.
WHY THE EXCITEMENT?
LCA is an inherited retinal disease that causes vision loss at an early age due to a mutation on the RPE65 gene. It affects about 3,000 people in the United States and there is currently no approved treatment for this condition. This gene therapy doesn’t just delay loss of sight but it can actually improve it. Just imagine what this could mean for those with LCA? Joy for starters. People from the clinical trials spoke of seeing their mother’s face for the first time, seeing stars in the night sky, and seeing snow and rain falling.
FFB’s new CEO Dr. Ben Yerxa also marvels at what a difference this treatment could make, if approved. He said, “This is a therapy that really improves vision…that is a remarkable thing. That shows us it’s possible… that if we have the right target and the right approach we can make a huge difference on peoples’ vision.”
WHERE DO YOU AND TWO BLIND BROTHERS FIT IN?
It is important to us that everyone understands the tangible effect of buying our shirts. The funds from Two Blind Brothers are driving life-changing treatments like this one. When you buy our shirts you become part of the cure. Our goal is to find those researchers who have a great hypothesis, a little bit of a proof of concept and who are trying to get ready for a clinical trial. We fund promising early clinical work that others don’t always have the temerity or money to do so. We want to give these projects wings to get going. Some of the early research for Spark’s therapy was funded by the Foundation Fighting Blindness, an organization we work very closely with./p>
WHAT ARE THE NEXT STEPS
While today’s vote is non-binding, meaning that the FDA does not have to follow the recommendations of the advisory committee, it generally does heed their guidance. The advisory panel is expected to give their two thumbs up. The FDA will then have until January 12, 2018 to give a final ruling. If (and hopefully once) approved, discussions around pricing begin.
Let’s go for goal!