Eliza was first child ever to receive gene therapy for Childhood Alzheimer’s
The O’Neill family’s fight with Sanfilippo Syndrome, known as Childhood Alzheimer’s, has been long and tough, but they aren’t backing down anytime soon. After receiving a miracle clinical trial for their little girl Eliza, the family’s focusing their energy on helping other kids just like Eliza receive the new gene therapy. “There are many more children than spots available. We can’t leave the others behind to suffer and die,” Eliza’s dad, Glenn ONeill, told Scary Mommy.
Two years ago the South Carolina family saw their 4-year-old’s mind deteriorating. After finding out about a clinical trial for Sanfilippo Syndrome at the Nationwide Children’s Hospital in Columbus Ohio, the family started fundraising. Their community efforts in person and online brought in about $250,000, which was great but not nearly enough. Benjamin Von Wong joined the fundraising efforts by creating a video for the family that eventually went viral and led to $2 million in donations for the gene therapy trial.
After raising money for Eliza and two other kids who received the clinical trial, the O’Neill family did another incredible thing and went into isolation for 726 days. “We chose to protect Eliza from one particular virus that might exclude her from the trial. It was a long, tough road, but we made it,” Glenn told us. “The only people to consistently enter our home were Eliza’s therapists who were dressed head to toe in protective gear.” Since he works from home the family was able to consider total isolation to protect Eliza. His wife, Cara, quit her job and their son, Beckham, completed school online. The family sanitized everything that came inside their home and relied on friends and family for mail, grocery store, and bank trips. “When we thought about an exclusion criteria for Eliza’s only chance at life, we made the decision,” Eliza’s dad shared. “We never thought it would last 726 days.”
Fast forward to this May and little Eliza was the first child in the world to receive the new gene therapy for Sanfilippo Syndrome. “We do see a new light in her eyes and a new connection with us. We thought this was lost. She is much more engaging in activities. The other day, she brought a small toy specifically to us to play with together, which hasn’t happened in a very long time,” Glenn shared with us. Eliza lost various skills because of the disease, including her ability to speak. But things are looking better since the gene therapy. “You can tell she wants to learn more. She is trying. She is in there, and she’s doing it all with a smile on her face!”
Since then the family has recommitted themselves to raising money to help other kids fight Sanfilippo Syndrome. Glenn thinks the gene therapy could help many more kids. “We are very, very, hopeful,” he said. “In fact, early indications are that the gene therapy may indeed be having a positive effect.” Thanks again to the help of Benjamin Von Wong they’ve got a new video to help raise awareness and funds for more clinical trials. “This is a stepping stone which opens up doors for every child out there, depending on the results,” Glenn explained. “Shouldn’t every child be afforded a chance at life?”
The goal now is to raise as much awareness and money as possible so the O’Neill family can help other families across the country who have little ones with Sanfilippo Syndrome. “Eliza’s legacy is in helping her friends to receive a similar chance that she did,” her dad shared. “Miracles can happen for these little one’s, but the time has to be now.”