A Utah toddler is one of the first in the U.S. to receive a potentially groundbreaking therapy, and his father is doing everything he can to give his son the strength to win his fight.
Deep in the farm country of central Utah, a family is counting their blessings.
“Yes, I would call it living the dream, yes ma’am,” said Alex Wight, who lives with his family in Moroni.
Alex Wight reads the book he wrote about courage to his son, Cinch.
Four years ago, while riding in the high country, Wight and his horse went off a cliff. The fall shattered Wight’s foot. Far from help, “I was definitely going into some shock and I thought, ‘Man, I’m never going to get out of here,’” Wight said.
Wight climbed back on that horse and rode for 20 miles. “If I would have quit, I would have never made it out,” he said.
Never was that toughness more vital than when his wife gave birth to their son, Cinch. “I was pretty torn up. I cried a lot. Not gonna lie,” Wight said.
Newborn screenings showed Cinch has spinal muscular atrophy, or SMA.
“I cried. I was working at the sawmill, didn’t even tell anyone I was leaving, (I) just put my machine away and left,” Wight said.
The neuromuscular disorder paralyzes a baby in the first few weeks or months of life.
“My first thought was that my son will never be able to ride broncs, rope, or just be a cowboy in general,” Wight said.
Many babies with SMA don’t survive to their second birthday, but the day after Cinch was born, the U.S. Food & Drug Administration approved a new therapy. “We felt like our prayers were answered,” Wight said.
In a single infusion, a virus delivers a new copy of the gene into the baby’s nerves, said Dr. Russell Butterfield, a pediatric neurologist with University of Utah Health and Intermountain Primary Children’s Hospital.
“Only this time, it’s not delivering a diseased gene that just causes more virus to be made, but a gene that we care about,” Butterfield said.
It’s a $2.1 million treatment.
“I was thinking, ‘Man, there’s no way I’ll ever pay for it,” Wight said.
Wight was determined. “We just stayed on top of it. I wasn’t going be the one to not get it,” he said.
With help from insurance and Primary Children’s Hospital, Cinch got the gene replacement therapy through the Primary Children’s Center for Personalized Medicine.
The Wights wouldn’t know for months if it worked. Amber Wight, Cinch’s mother, said, “Every day’s a waiting game, we have no idea.”
Alex wrote a book to teach children they can do hard things, even when they’re hurt or discouraged.
“Early one morning, a cowboy stepped out of his camp and into the corral,” Wight said, reading the book to his son.
He held his breath the day Cinch took his first steps. “He just cruises,” Wight said.
“It just brings a tear to your eye, to be honest,” Butterfield said.
Time will tell if the gene replacement therapy is indeed a breakthrough. “I just hope he never quits,” Wight said.