With drug companies reluctant to spend research money on rare diseases, parents of children suffering from such ailments often feel the burden or raising funds and awareness of these diseases themselves in an effort to save their own children’s lives.
That’s exactly what the parents of four-year-old Eliza O’Neill of Columbia, S.C., are attempting to do with the viral video above about her condition, Sanfilippo syndrome, a degenerative disease that destroys brain cells and kills its victims usually in their teenage years. So far, there is no cure for this rare neurological disorder.
Cara and Glenn O’Neill told “Today”
that if their daughter does not get an experimental treatment, she would stop speaking by age five or six, and then lose her ability to walk and other motor functions as she continues to regress as a result of the disease.
The O’Neills are counting on research being conducted at Nationwide Children’s Hospital in Columbus, Ohio, where scientists have discovered a gene therapy that cured the disease in mice. “Today” reports, “The researchers were ready to start testing the therapy in kids, but they needed to raise $2.5 million to put together a clinical trial. And because Sanfilippo is rare, no drug companies were signing on to fund it.”
With the help of Canadian filmmaker Benjamin Van Wong, who volunteered to help, the O’Neills created this moving video and have now raised more than $380,000 in six months for the clinical trial.
But as “Today” reports, “Even if the O’Neills manage to raise the $2.5 million, they know it doesn’t guarantee Eliza will be helped. She may not qualify for the trial, though researchers think she probably will. The trial may not work. And there are always risks with any unproven therapy. With no other treatments available, those risks seem minor when weighed against the certain death that awaits Sanfilippo kids.”
“As a parent, you have to be the advocate for your child,” Cara O’Neill told “Today.” “You have to give them the best shot possible. And this is her best shot.”