When the O’Neill family learned that their daughter Eliza, then 4, might qualify for a clinical trial for gene therapy for her rare, life-limiting condition, they knew they would do anything to help her.
What did anything entail? Isolating themselves from the outside world for 726 days — four days short of two years — to protect Eliza from contracting a harmless virus.
“My son, Beckham, my daughter, Eliza, Cara, my wife, and myself never would come in contact directly with any people or anything that people touched,” O’Neill, of Columbia, South Carolina, told TODAY. “We had success in not getting sick at all.”
The O’Neills are sharing their experience as others around the world are facing quarantine or isolation due to possible coronavirus exposure.
“If we can do it for 726 days, 14 days is possible,” O'Neill said. “It's very different why we were doing it versus why someone with coronavirus would do it. I would think the people that are doing it have the opportunity to get better hopefully and then go back out in public after 14 days.”
Eliza, now 10, has Sanfilippo syndrome type A, a rare neurodegenerative disease that is sometimes referred to as childhood Alzheimer’s. Children with it progressively lose their ability to speak, understand and move, and most of them do not survive their teenage years. Type A is the most severe form of the condition, which has no treatment or cure.
When the family heard of a clinical trial of gene therapy for the disease, they made the “Saving Eliza,” video that raised $2.1 million for research. The couple also runs the organization, Cure Sanfilippo Foundation, which has raised $8.5 million to support more than 20 research projects worldwide.
“We are fighting for treatments and hopefully a cure for Sanfilippo,” O'Neill said. “You don't fund research for one child. It's for all these children.”
While they never knew if Eliza would even qualify for the trial, they knew she couldn’t be part of the gene therapy if she caught the harmless virus AAV9. The virus was the vector that delivered the gene therapy and if the body developed immunity to it, it wouldn’t work.
“We were worried that if she catches this virus, we won't even know if she does, so we might have ruined it all,” O'Neill explained.
They decided to isolate themselves. At the time, they had no idea how long it would be and they thought it could be anywhere from three to six months.
“There was no going back. We were protecting her from a virus and by protecting her we had to protect ourselves as well. That's why all four of us had to do it together,” O'Neill said.
One reason they needed to isolate is that children with Sanfilippo often put their hands in their mouths, making it almost impossible to keep them germ-free.
“We knew there was no way we could prevent her from doing this,” Dr. Cara O’Neill, Glenn's wife, told TODAY. “She wasn’t going to follow the normal don’t touch your mouth.”
(It appears Eliza's not alone in struggling to keep her hands from her face. Many adults are now realizing that avoiding touching the face is challenging.)
726 days of 24/7 contact with your family
Some things were easier to plan than others. Cara’s mother bought groceries and left them on the porch. Then they cleaned everything with disinfecting wipes before bringing into the house. They ordered as much as they could online and stocked up on gloves and masks. Friends opened mail and visited the bank for them. O'Neill worked remotely and Cara, who is a pediatrician, stopped working in a clinical practice. The kids used the online public school platform for their education.
“We love each other but imagine 726 days with three other people. You never leave each other. I mean it nearly will drive you crazy,” O'Neill said. “We just we got creative.”
That means Beckham enjoyed a lot of virtual play dates, they hosted indoor picnics, the kids drew on the walls and they held dance parties, including a daddy-daughter dance for Eliza and O'Neill on Valentine’s Day so she had the same experience as her classmates. They even went on isolated day trips to a beach and a field that people never visited.
“It's boring but that was our place,” O'Neill said. “We would kick the ball around the four of us or we would come up with little games.”
Four days short of two years of isolation, they learned that Eliza qualified for the trial and she became the first patient with Sanfilippo in the U.S. to receive gene therapy in 2016. The dose Eliza received is significantly less than what participants later received. While she can't speak any more and she needs constant care, she still has some motor skills.
“She can use a fork in her food and still eat it. She can still swallow. She can still walk. She can still run short distances,” O'Neill said. “She's kind of holding her own.”
The family remains optimistic for the future.
“We hope she has more good days,” Cara said.
CORRECTION (March 12, 2020, 1:30 p.m. EDT): An earlier version of this article misstated the virus that could have harmed Eliza. It was AAV9, not AA9V.