Eliza O’Neill, 5, suffers from Sanfilippo Syndrome (Type A), a fatal progressive disease that primarily affects the brain of children born with this disease. However, Eliza’s parents, Glenn and Cara O’Neill, have not given up hope for their daughter. The family has placed themselves on a voluntary quarantine in order to give Eliza the best chance at being offered a spot for clinical trials. The O’Neill family has been quarantined for over 280 days without physical contact with people outside the home. Even items shipped to the home and groceries delivered to their doorstep by Eliza’s grandmother are disinfected before entering their living space.
The Daily Mail reports that the isolation is Eliza’s best bet at being offered a place in clinical trials should they come open.
“We thought we’d be closer to a clinical trial start date by this point. As things stand, even if we get news of success in the drug making soon, we are still likely at least six months away. We won’t ever give up though.”
According to Cure Sanfilippo Foundation, Eliza’s condition is dire and her family has decided to sacrifice their freedom for their precious daughter in exchange for the hope that she may be able to win the battle with the deadly disease. Children with Sanfilippo Syndrome typically only live to their mid-teens. Meanwhile, they progressively lose the ability to talk, walk and function on their own.
“Children with Sanfilippo Syndrome typically do not show signs of this devastating disease until 2-6 years of age. The disease is working on them from the time of conception. However, it takes time for the body and brain to accumulate enough waste material to begin to cause symptoms like hyperactivity, speech delay, autism, recurrent ear/sinus infections. Children then begin to lose the skills they used to have- talking, walking, eating. Most children develop seizures and painful movement disorders before they pass away. Typical life expectancy for the most rapidly progressive form of Sanfilippo Syndrome (Type A) is in the mid-teens.”
Eliza’s mother, Cara, was a special needs pediatrician before moving to isolation with her daughter. Therefore, Eliza is in great hands. Though most children with Sanfilippo Sundrome begin to lose their ability to speak at around age four or five. However, five-year-old Eliza has retained her ability to count and speak, though she is beginning to have difficulty. Therefore, the family is hoping for a clinical trial soon. The O’Neills raised over $2.2 million in funding for the Cure Sanfilippo Foundation and are hoping researchers pull through soon with a clinical trial option.
“Multiple teams are working on it, but to a parent of a child now five years and three months with Sanfilippo, time is not on our side.”
Why is isolation so important for Eliza to participate in clinical trials? Glenn notes that the quarantine hinges on ensuring his daughter does not come into contact with AAV9 virus or build an immunity to it as it will be used to deliver the drug product.
“But, if Eliza were to build up immunity to the AAV9 virus that they are using to deliver the drug product, she would not be selected for the trial…her only chance at life.”
Therefore, Glenn says the family has no plans of coming out of isolation until a clinical trial is within sight. Though the family is isolated from the rest of the world, they don’t want their daughter to miss out on the small things in life, such as the Frozen-themed dance at the local school. Therefore, the O’Neills recreated the school dance, complete with decorations and music, in their own home for Eliza. Watch the heart-warming video above to see Eliza’s reaction to her personal dance party.
You can follow Eliza’s battle with isolation and Sanfilippo Syndrome on the family’s Facebook page, Eliza’s Story.