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[text on screen] An Interview with Amber Salzman, Ph.D. President, The Stop ALD Foundation. Amber Salzman comments on Science publication describing gene therapy success for adrenoleukodystrophy (ALD).
[text on screen] What are you so excited about?
Amber Salzman: I've seen ALD up close. I have a nephew who passed away from ALD. I have another nephew who had ALD but was treated with a stem cell transplant.
He had very bad GVH from the transplant and he's now confined to a wheelchair.
My son was born with ALD. He was one of the lucky ones. He had a very successful transplant and he's a healthy 9-year-old today.
So, I've seen it in pretty bad ways: boys who died from it, who didn't have matches, and boys who the transplant itself left fairly disabled.
So, to hear about two boys who were cured from this treatment, and a third one, recently treated and doing very well, brings some comfort to the pain felt in my family.
[text on screen] Why is this scientific development important?
Amber Salzman: Gene therapy has been talked about for some time. However, there have been setbacks, and many have been discouraged to use gene therapy.
So, to see boys cured by it, I think really opens the door to use this technology to better help other families afflicted by severe diseases.
[text on screen] What role did The Stop ALD Foundation have in getting to this point?
Amber Salzman: The Stop ALD Foundation had a vision back in 2001 as to how gene therapy could be used in a treatment for ALD.
And we were relentless in bringing resources to bear and removing any obstacles preventing us from achieving that vision.
We talked to the ALD experts around the world, gene therapists, the manufacturing companies, regulatory agencies, all the various disciplines it would take to actually pull this treatment off.
And some people said they couldn't help us, but we just didn't take no. We explained to them what they could do and why it made sense for them to help us and what this could do, not just for ALD, but future diseases that afflicted way too many people.
[text on screen] What role will The Stop ALD Foundation have going forward and how will you accomplish your goals?
Amber Salzman: It's very important to us that this trial be initiated in the U.S., because it's only available in Europe right now.
So we want a lot more patients to have this capability available to them.
It will take more resources; resources we don't have. We're going to try to leverage what's out there.
[text on screen] What message would you like to share with patients and loved ones affected by ALD?
Amber Salzman: There's research going on for patients in all stages of ALD. Our foundation, The Stop ALD Foundation, is firmly committed to bringing more therapies to bear for these patients.
The other thing I would say is please share this information with everyone you know. We really need more focus, more attention, and more resources to have an impact in terms of helping all of our loved ones.
[text on screen] Produced by The Stop ALD Foundation, www.stopald.org. For more information please contact Amber Salzman, ph: +1.610.659.1098 * amber@stopald.org