Great Urgency

July 15, 2015

by Mark Dunning

I saw my friend Carol recently. She was traveling with her seven year old daughter Sara. Carol has Usher syndrome and her vision is fading. She will soon lose what is left of it. There will be a day in the not too distant future when she will see Sara smile for the last time. And given that she lives overseas, it is quite likely that when we part it will be the last time she sees my face.

I don’t often feel like crying these days. More often I feel more like dancing. We are going to have treatments for Usher syndrome. They aren’t just ethereal ideas. I have SEEN them. All the parts exist TODAY. My fingers are shaking with excitement as I type this. I can’t use enough capital letters for emphasis. WE ARE GOING TO CURE USHER SYNDROME!

Today, right now, this second, we have the technology to perform gene therapy. We have viruses with the capacity to hold every Usher gene. There is a gene therapy trial going on RIGHT NOW on Usher syndrome type 1b. It’s just a matter of putting the pieces together.

Today, right now, this second, we have the technology to create retinal cells from stem cells. We have methods for delivering the stem cells. And with CRISPR technology, we can even edit the flaws in the genes in those stem cells. It’s just a matter of putting the pieces together.

Retinal implants and cochlear implants are already FDA approved, but today, right now, this second, we have the technology to create a combined vestibular and cochlear implant. There are clinical trials going RIGHT NOW on vestibular implants. It’s just a matter of putting the pieces together.

With gene therapy we have the technology to stop the progression of the disease. With stem cells we have the technology to replace damaged cells and restore vision. With vestibular implants we have the technology to improve balance the way we have improved hearing with cochlear implants. 

It is all there. It’s just a matter of when. But just because we are going to succeed doesn’t mean we shouldn’t proceed with the utmost urgency. The rules have changed. We no longer have to hope that someday there will be treatments. There will be. So it’s time to dream bigger than just treatments. It’s time to dream about treatments that come in time.

I want to look in to Carol’s eyes this last time and I want to tell her, with certainty, that she will see me again, literally see me again, some day. Stem cells are coming. Improved retinal implants are coming. But I don’t want them to come for the next generation. I want them to come for Carol. I want Carol to see Sara’s graduation. I want Carol to see Sara’s wedding.

Image of starry sky with quote "I want to tell the parents of a young boy named Hunter that he will forever see a thousand stars in the sky, not just a handful..."

I don’t want children born today to miss the stars. I want to tell the parents of a young boy named Hunter that he will forever see a thousand stars in the sky, not just a handful. I want Hunter to drive a car at sixteen years old. I want Hunter to have no limitations, to have the opportunity to be a fighter pilot or an architect or a surgeon when he grows up. I want that for Hunter, not the next generation.

And I desperately want my friends with Usher who have already lost their vision to see me, finally really see me. I want my friend Moira to recognize me from a distance. I want her to take her guide dog Finnegan off the harness forever. I want her to go for walks while Finnegan, permanently retired, romps around her.  

There is an incredible opportunity before us. These dreams are possible. We just need the right sense of urgency, the right amount of funding, and the right amount of help. If you have the means, please help us out.  For those of you willing to participate in fund raising efforts, watch this space for our upcoming Usher syndrome awareness event.

And Carol, Hunter, and Moira, keep believing. We’re coming as fast as we can.

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