After raising more than $1 million in the span of months, Eliza O’Neill’s family is closer than ever to funding a clinical trial to save the 4-year-old from Sanfilippo syndrome, a rare neuro-degenerative disease that, if left untreated, will soon rob Eliza of her speech, her ability to walk and, ultimately, her life. But they’re still more than $600,000 short of their online fundraising goal and their fundraising deadline looms at the end of the month.
“In order to get the funds to where we can be confident that the trial will be fully funded for all children to participate, we have set the goal at the end of October,” Eliza’s father Glenn told me in an email. “You have to draw a line in the sand, and when it’s your child’s life on the line, you don’t want to cut it close.”
After starring in a viral video last spring and breaking GoFundMe.com’s fundraising record through their Saving Eliza campaign last June, the South Carolina family now hopes that a month-old fundraising push modeled on the hugely successful ALS Ice Bucket Challenge will work wonders. The #Sing2Lines campaign asks participants to record themselves singing or lip synching two lines of a song, challenge five friends to do the same and post the video online. Donations related to the campaign — participants are asked to give $50 each — helped generate an additional $75,000, with everyone from kids to police officers to a beauty pageant queens to actress Andie McDowell taking part.
“ALS made history,” Glenn O’Neill said. “We don’t need to make history, but do need help.”
Though the O’Neills have made amazing strides in raising both awareness of and money for Sanfilippo syndrome, the fact that the disease is so rare — affecting just one in 70,000 births, according to the government — has made their fight that much harder. Forbes reports that fewer than 500 drugs are approved by the FDA to treat some 7,000 rare diseases.
The treatment that the O’Neill family is counting on is expected to win approval for a clinical trial from the FDA in early 2015. The treatment is a promising gene therapy created by researchers at Nationwide Children’s Hospital in Columbus, Ohio and, if all goes as planned, it will be administered starting this February to nine children, including Eliza. Nine more children may be treated at an additional site in Europe.
“If it acts in humans as it did in the animal models, it is a miracle,” O’Neill said. “But we won’t know that until we fund this trial.”
The O’Neill family’s nonprofit foundation, the Cure Sanfillipo Foundation, in the meantime has also backed and funded the grant writing for a $1.5 million grant from the National Institutes of Health, but it remains questionable whether the NIH, which saw its budget slashed last year, will ultimately have the money to spare.
“We go on fundraising as if this is not coming because until we see the check being made out for the trial, we can never be sure,” O’Neill said.
While they work to raise funds, the O’Neills also work to help Eliza maintain her current skills. A video posted in August shows her singing the song “We Are Young” by the band Fun.
“She is so inspiring and brave. I whisper to her every night at bedtime how incredibly proud we are of her,” her father said. “She isn’t even aware of how strong she is.”
Image courtesy of www.savingeliza.comMore On