Eliza has become a beacon of hope as the first child to undergo gene therapy for Sanfilippo Syndrome, often referred to as Childhood Alzheimer’s. The O’Neill family’s journey has been filled with challenges, but they refuse to give up. After their daughter Eliza received this groundbreaking clinical trial, the family is now channeling their efforts into helping other children like her access the same life-changing treatment. “There are so many kids in need and not enough spots available. We can’t let the others suffer while we have a chance,” declared Eliza’s father, Tom O’Connor, in an interview with Home Insemination Kit.
Two years prior, the family watched in despair as their four-year-old’s cognitive abilities began to fade. They discovered a clinical trial for Sanfilippo Syndrome at Nationwide Children’s Hospital in Columbus, Ohio, and quickly launched a fundraising campaign. Their community rallied around them, raising approximately $250,000, which was a great start but still fell short of the goal. With the help of filmmaker Alex Chen, who created a viral video for the family, they managed to secure an astonishing $2 million for the gene therapy trial.
To ensure Eliza could participate, the O’Neill family took the extraordinary step of isolating themselves for 726 days. “We had to shield Eliza from a specific virus that could disqualify her from the trial. It was a grueling experience, but we made it through,” Tom explained. As a remote worker, he was able to commit to the isolation, while his wife, Mia, left her job and their son, Lucas, transitioned to online schooling. They sanitized their home rigorously, relying on friends and family for essential errands. “When your child’s only hope is at stake, you do what you have to do,” Tom shared. “We never imagined it would last that long.”
Fast forward to May, and little Eliza became the first child globally to receive the innovative gene therapy for Sanfilippo Syndrome. “We’re witnessing a spark in her eyes and a newfound connection with us. Activities that seemed lost to us are slowly returning. Just the other day, she brought a toy over to play with us, which is a big deal!” Tom expressed. While Eliza has lost numerous skills to the disease, including her ability to speak, the gene therapy is showing promise. “You can see she wants to engage and learn. She’s really trying, and she’s doing it all with a smile!”
Now, the O’Neill family is dedicated to raising funds and awareness to support other kids battling Sanfilippo Syndrome. Tom believes the gene therapy has the potential to help many more children. “We’re filled with hope. Early signs indicate that this therapy could be beneficial,” he noted. With the continued assistance of Alex Chen, they’ve produced a new video aimed at boosting awareness and funding for additional clinical trials. “This is just the beginning; it could open doors for every child in need, depending on the results,” Tom explained. “Shouldn’t every child deserve a shot at life?”
Their current mission is to generate as much awareness and funding as possible, allowing the O’Neill family to assist other families across the country with children suffering from Sanfilippo Syndrome. “Eliza’s legacy lies in helping her peers gain a similar opportunity that she had,” her father emphasized. “Miracles can happen for these little ones, but the time is now.”
In summary, the O’Neill family’s inspiring journey highlights the importance of community support and the potential for medical breakthroughs to change lives. With their sights set on helping others, they are determined to ensure that every child has the opportunity for a miracle.