Family's campaign for $1 million to save daughter suffering from rare condition goes viral

[Oceander]

Daily Mail

Family's campaign for $1 million to save daughter suffering from rare condition goes viral

By Daily Mail Reporter
Published: 23:05 EST, 2 April 2014 | Updated: 23:12 EST, 2 April 2014

Eliza O'Neill is just four-years-old but in six months, she will stop speaking.

In the next four years, the energetic child won't be able to walk or feed herself, before developing seizures and movement disorders. And then, she will die.

But the South Carolina toddler's family hope to avoid the horrible prognosis by raising $1 million to fund a known cure for Sanfilippo Syndrome, the disease Eliza was diagnosed with in July.

'You know where it's headed. It's headed towards pain for her and as a father you want to be able to protect your children,' Eliza's devastated father, Glenn O'Neill, said.

'The donations that come in will fund this clinical trial and fund a chance at life for Eliza. That is the hope we hold on to.'

New York Daily News reported Nationwide Children's Hospital in Columbus, Ohio, plan to test the cure, but it will cost about $2.5 million to produce the treatment and run the trial.

The gene therapy trial scheduled for late 2014 is specifically for children with Sanfilippo Syndrome, a rare metabolic disorder that causes major muscle growth deficiencies.

Eliza's parents, Glenn and Cara O'Neill, say the treatment would save their daughter and literally stop the disease in its tracks overnight.

They're in a desperate race against time to raise enough money to fund the cure that would save Eliza and others like her.

The O'Neill's say Eliza is 'a fun loving four-year-old who loves to sing, run and most of all, cuddle. She loves to play dress up and horse around with her rowdy big brother Beckham'.

The Dora the Explorer-loving toddler is also incredibly active and loves to spend time with her family.

But that could all change if her condition isn't treated in the next six months.

'We have never heard of another child [with her condition] that reaches five-years-old and is still speaking,' Glenn O'Neill told Daily News.

'We have the cure. It's going to happen at some point. But it won't happen without money and if it gets delayed, Eliza won't get it and a lot of other kids won't get it.'

The family began raising money in their community six months ago, collecting about $250,000 through fundraisers and other events.

But with time against them, they decided to amp up their efforts.

The couple did a Google search on 'how to make a viral video' and connected with  filmmakers DL Cade and Benjamin Von Wong, who produced the film for the family: 'Be part of the miracle: Saving Eliza', available on YouTube and on a GoFundMe fundraising page.

Thanks to the video, the O'Neill's have raised nearly $60,000 on GoFundMe, but they are well off their target.

'Hope is a nice word, but we need action,' Cara O'Neill said.

'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...

'It's not a question of if this can happen. This will happen.'

Sanfilippo Syndrome is caused by a defect in a single cell that eventually leads to progressive degeneration of the central nervous system.

Most sufferers don't live past their early teens, and don't show visible signs until their preschool years.

CNN reported that Sanfilippo has been overlooked by the pharmaceutical industry because it is so rare.

About 16 children suffer from Sanfilippo Type C in the United States.

This means very few federal dollars are allocated for funding rare disease research.

However, actor Jonny Lee Miller has actively campaigned for more funding for the disease since learning a crew member's son, Jonah, was diagnosed with Sanfilippo Syndrome.

'The answers and the cures are right around the corner,' Miller told CBS.

'There’s just not enough money going into this right now, and we’re talking about little kids.'

Miller has addressed a congressional caucus in Washington on behalf of rare disease organizations.

He also founded the Jonah Just Begun Foundation and will be running in the North Face Bear Mountain Run in Bear Mountain, New York on May 4 and 5 for Jonah and for Jonah Just Begun.

To help fund a cure for Sanfilippo Sydrome, visit GoFundMe - Eliza O'Neill or Facebook - Eliza's Story.