WATCH: Eliza O’Neill’s Family Expresses Hope, Excitement Post-Treatment
COLUMBIA, S.C. (WOLO) – Just shy of two years in seclusion, and Eliza O’Neill’s family received the news they’d been waiting by the phone for. Their daughter, who has Sanfilippo Syndrome, became the first person in the world to be treated as part of a clinical trial.
The Forest Acres family’s story has been closely followed around the world.
“Since she’s been treated, there is a look in her eyes. It’s not all the time, but you get these moments of clarity from her, especially late at night when she’s in the bed and she knows it’s time for sleep and she’ll look at you when you’re reading a story and there is moments of clarity that something is changed,” her father Glenn O’Neill said. “Something is different.”
Glenn and Cara O’Neill’s daughter Eliza was diagnosed four years ago with Sanfilippo Syndrome, a rare degenerative disease that slowly robs children of their skills and knowledge until they die in their teenage years.
They raised millions to bring a clinical trial to life and went into seclusion to ensure that Eliza would not contract a virus that would disqualify her from it. On the 726th day, they got the call from Nationwide Children’s Hospital in Columbus, Ohio. With a one-time dose given through an IV in her hand, Eliza became the first patient in the world to receive the gene therapy.
“Eliza; the last two years have been very, very rough, but she still can say words, she still has understanding and we’re so thankful for that,” Glenn said.
Since that 30 minute treatment on May 10, the family has been readjusting to life outside their home. No longer limited to Skype sessions, Eliza goes to the Autism Academy and the Therapy Place. Her brother Beckham heads back to school in the fall.
“It’s pretty surreal that feels like an eternity for us from when we began,” Cara said.
“Every single dollar mattered, and I just hope that everyone watching that has followed our story, that has donated, that has helped, realizes how thankful we are for that, because that literally allowed us to have hope for Eliza, and other children as well, are being treated today with this.”
And just last week, some positive initial results for the gene therapy. The update shows reduced toxic buildup levels in Eliza and the other patient who was treated.
The O’Neill’s are taking it day by day, incredibly grateful for any small signs of improvement from their daughter. They’ve received a chance for Eliza. Now, their focus shifts to the countless other sons and daughters who need the same shot.
“The family the community, they are our family to us now,” Cara said. “And we can’t leave them behind. We know that feeling and it’s not right.”
On Wednesday night at 11, you’ll meet some of the other children who need that chance in part two of our series.
To learn more about the Cure Sanfilippo Foundation, click here.