Saving lives with gene replacement therapy
Gene replacement therapy. It’s a game changer when it comes to treating life-threatening illnesses. It can replace disease-causing genes with healthy genes, knock out a gene that’s not working right, or add a new gene to the body to help fight disease. To date, the FDA has approved four types of gene therapy including one that was given the OK just in time to save one little boy’s life.
No doubt about it, Cinch Wight is going to be a cowboy just like his dad.
“He loves the dog and the horses and the cows,” shared Cinch’s dad, Alex Wight.
But it has been a wild ride for this young bronco. A mandatory newborn screening test at birth revealed Cinch had spinal muscular atrophy or SMA.
Cinch’s mom, Amber Wight recalled, “That was the first time I’d ever even heard the term and what it was. And so, it was very scary.”
A neuromuscular disorder that can paralyze a baby in the first few weeks of life.
“My first thought was, he’s never going to be able to ride broncs or anything like that,” expressed Alex.
But just one day after Cinch was born, the FDA approved a new gene therapy.
“We were pretty excited to get a phone call from the department of health, you know, and have this baby here who we can use this treatment on after its approval,” explained Russell Butterfield, MD,
pediatric neurologist at University of Utah Health and Intermountain Primary Children’s Hospital.
A critical gene in little Cinch was missing. Pediatric neurologist Russell Butterfield used an infusion to deliver a virus carrying a new copy of the gene into Cinch’s nerve cells.
“It’s like a delivery truck to deliver genes to where you want them to go. What that does do, is it stops the disease right where it is,” elaborated Dr. Butterfield.
Just a few years ago, most children born with SMA didn’t make it to their second birthday. Now?
“The hardest is holding a baby in one hand and holding that drug in the other and really feeling the weight of that. And understanding that how different this child’s life will be with this new medicine,” expressed Dr. Butterfield.
It took a lot of courage for this family to get this far. That’s why Alex wrote a book for his son. A true story about how real cowboys never give up.
“I wanted to let him know that no matter how hard it gets, as long as he keeps going, he’ll be all right,” shared Alex.
Doctors don’t know if the one-time infusion will last a lifetime or will have to be repeated and there could be a possible risk of inflammation to the liver that doctors will closely monitor. The gene replacement therapy costs 2.1 million dollars. Insurance paid for most of it, but Alex hopes sales from his children’s book will help pay the rest. You can find the book, A Cowboy and His Horse, at https://www.amazon.com/COWBOY-HIS-HORSE-ALEX-WIGHT/dp/B08CWG46ZX.
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