4-year old Eliza O'Neill will likely start to lose her ability to speak, walk and interact normally with others by the time she turns 5 years old as her rare genetic disorder starts to destroy her brain cells and cause irreversible brain damage.
Eliza's rare metabolism disorder known as Sanfilippo syndrome is characterized by the lack of an important enzyme needed for normal cellular function. Children who have the condition accumulate a toxic material in their brain and body overtime resulting in severe disability and death. The disease only affects about one in 70,000 individuals but those affected have slim chances of surviving beyond their teen years. Eliza has Sanfilippo type A, the most severe type of Sanfilippo syndrome putting her at high risk of dying at a very young age.
To date, there are no available treatments for the disease albeit researchers from the Nationwide Children's Hospital in Ohio have successfully tested a new gene therapy for Sanfilippo syndrome on mice. Despite the promising results on animals, the researchers could not proceed to testing the therapy on humans because launching a clinical trial that would test the gene therapy on children with Sanfilippo syndrome would cost $2.5 million.
Desperate for a treatment that could potentially save their daughter's life, Cara and Glenn O'Neill turned to the internet for help so they can raise enough funds for the trial. Although there is no guarantee that the experimental treatment would indeed work or if their daughter could make it to the clinical trial, the couple knew they needed to do something to help save their daughter.
The O'Neills set up a fundraising raising campaign on Gofundme.com and aided with viral videos that raise awareness about Eliza's condition. The couple started to receive financial help from complete strangers, and in just a few months, the "Saving Eliza" campaign has raised over $820,000 and with about $250,000 donations from their local community, the family has already raised more than $1 million for the clinical trial that could potentially help not just Eliza but other children with Sanfilippo syndrome as well.
Although the family still needs to raise about 1.5 million, Glenn O'Neil said that the generosity of total strangers has made them hopeful that they will be able to raise the needed amount of money.
"We thought money would be a hurdle we couldn't overcome," he said. "Now though, if $1 million can be achieved in such a short amount of time, then why can't it happen again?"
