Ready, Set, Scheduled. Florida Boy Will Receive Gene Therapy Treatment to Reverse Vision Loss
This is the second in a series following the progress of Creed Pettit, a 9-year-old Florida third-grader, who completed treatment in March with the breakthrough gene-therapy drug called LUXTURNA™, approved as the first gene therapy for RPE65 genetic mutations and as the first-ever genetic therapy in the United States for an inherited disease.
Sarah St. Pierre Pettit and her son, Creed, made the six-hour trip to a Miami eye hospital many times in the past, but on their most recent visit, driving back home was different.
“We usually have hope when we leave from Miami,” said Sarah, referring to hope for a treatment for her 9-year-old son’s vision loss due to Leber congenital amaurosis.
“This time was the first time I’ve ever come back home from Miami with a set date and hope.”
The next important date will be March 28th for the same surgery in Creed’s other eye.
“I am on CLOUD 9!!!” Sarah emailed Monday, Feb. 19, hours after meeting with a team of doctors at Bascom Palmer.
The news comes after Sarah’s insurance provider, working with the developer of LUXTURNA™, Spark Therapeutics, gave the go-ahead to schedule surgery. The company established Spark Therapeutics Generation Patient Services™ to support commercially insured patients and their caregivers in the United States and help them navigate the insurance process, according to a Spark Therapeutics’ news release.
Soon after doctors diagnosed her son at the age of 3, Sarah began raising money, totaling about $100,000 that has gone toward research into finding a treatment for LCA-RPE65.
Creed, Sarah, Sarah’s friend, Chad, and Sarah’s mom, Mary, drove from Mount Dora to Miami on Sunday, Feb. 18; on Monday, a team of nurses and doctors met them and talked about what to expect for the surgery.
Meeting the gene therapy team
Dr. Audina M. Berracol will be doing Creed’s surgery. She is a Professor of Clinical Ophthalmology, specializing in areas including vitreoretinal diseases and surgery.
“She was amazing,” Sarah said. “We just had a chance to really meet her.”
Dr. Berracol answered Sarah’s questions and put her mind at ease. Creed has a sensory issue with anything that is “sticky” and had difficulty with patches on his eyes. To help overcome this, the doctor sent them home with a roll of tape used for patches.
“It’s to get him used to it because he’s not going to be able to pull it off,” Sarah said.
Creed later asked his mom how many minutes are in 24 hours. She Googled it and found 1,440 minutes.
He wanted to know because that’s how long he’s going to have to wear an eye patch after surgery.
“So that’s fine,” she said. “We’ll count down from there.”
Sarah also wondered what happens after surgery if Creed cries. It’s OK to cry, as long as he doesn’t rub his eyes. A small blister, called a bleb, forms after the surgery, creating vision as if looking through a fish-eye lens.
“Once it pops (naturally), you know you’re in the clear” the doctors said.
And Sarah asked about administering fluids intravenously, because Creed pulled out an IV after he woke up from having his tonsils out.
The doctor assured her that the IV will be placed so it can’t be pulled out.
She also learned the surgery usually lasts about an hour, much less time than she had imagined.
‘Excitement and worry’
Back home, Sarah reflected that everything was just so overwhelming.
“It was a long trip home but worth every second of it.”
Creed said the trip was “a one-night stay and we went to Bascom Palmer Eye Institute.”
As for the surgery, Creed said, “I’m feeling a little nervous.”
So is mom. Two days after returning from Miami, new fears crept in as she thought about all the “what ifs” that could happen during surgery.
“I can say other LCA moms are feeling this same emotion,” she said. “Waves of excitement and worry all flow through.”
Her feelings are smoothing out as she and Creed get back to their usual routine for the next several weeks before driving back to Miami for the surgery.
“We’re just going to plug along with school, and Creed’s school has offered to meet with me as to how we’re going to keep him on track while we’re there.”