A Columbia, S.C. family is reaching out to anyone who will listen in the hope of saving their 4-year-old daughter from a terminal disease.
"For two and a half, three years, I would say, we had the perfect, we had just the perfect life," Glen O'Neill said when talking about life after the birth of Eliza, Glen and Cara O'Neill's daughter.
Their perfect life was abruptly halted when doctors diagnosed Eliza with the rare, genetic disease Sanfilippo syndrome.
The disease kills brain cells and begins to take its toll on children around age 5 or 6. Over time, it leads to severe disability and eventually death, usually in the child's teenage years.
Getting the news that their affectionate, happy and active daughter had this condition was heartbreaking for the O'Neills.
"Right now Eliza is a fun-loving 4-year-old who loves to sing, run and MOST of all, cuddle," The O'Neills say on Go Fund Me. "She loves to play dress up and horse around with her rowdy big brother Beckham. She is, however, beginning to show signs of the disease in her learning and attention. And if nothing changes, it will only get worse from here."
According to the "Today" show, the change the O'Neills are hoping for lies in gene therapy from Nationwide Children's Hospital. When tested in mice, the treatment cured Sanfilippo syndrome.
Researchers are ready to begin tests for children, but they need to raise $2.5 million to organize the trial. Because Sanfilippo syndrome is so rare, drug companies were wary to fund it.
Although there are risks that the therapy won't work or participants will react badly to therapy, trials like these are sometimes worth the risks, Dr. James M. Wilson told the "Today" show. Wilson, director of the gene therapy program at the University of Pennsylvania, and his lab developed the viral vector being used at Nationwide.
“The possibility of an immune response is something that families clearly need to be aware of,” Wilson said. “But depending on how severely affected a child is, the benefits may outweigh the risks.”
The O'Neills became determined to raise the funds to give Eliza a chance at life. Irreversible damage begins around age 6, meaning Eliza does not have much time.
The family set out to create a video hoping to save their daughter soon. With the help of Canadian filmmaker Benjamin Von Wong and using the hashtag #SavingEliza, Eliza's story is spreading and funds are increasing.Comment on this story
Their Go Fund Me account has raised more than $504,000 in six months, halfway to their goal to raise $1 million through Go Fund Me before June 2014.
"Hope is a nice word, but we need action," Cara O'Neill says in the video. "We don't just have hope, there is something very real that exists that with enough money will happen. And will happen in time for our Eliza and other kids."
Alison Moore is a writer for the Faith and Family sections at DeseretNews.com. She is studying journalism and editing at Brigham Young University.
Email: firstname.lastname@example.org Twitter: @alison_kathleen