Saving Eliza: Family raising money for Sanfilippo Syndrome drug $530K away from goal

When Glenn ONeill and his wife, Cara, learned about a gene therapy treatment that could save their daughters life, they started a foundation and set off to raise the $2.3 million it would take to fund the manufacturing and clinical trial costs for the drug.

Today, thanks to about 26,000 donors from 70 different countries, theyre just $530,000 away from their goal.

Their daughter, 4-year-old Eliza ONeill, suffers from Sanfilippo Syndrome-Type A, a disease that causes children to lack an enzyme necessary for normal cellular function. The disease eventually causes a lethal buildup of a toxic material called heparin sulfate, leading to learning disabilities and behavioral problems. The disease is seen in 1 in 70,000 births, and most children born with with Sanfillipo Syndrome-Type A die by the time they are teenagers.

While there is currently no cure or treatment for the disease, researchers at Nationwide Childrens Hospital in Columbus, Ohio have found a potential gene therapy that, in a study, successfully rid mice of heparin sulfate buildup.

When Cara spoke to lead researcher Dr. Haiyan Fu, principal investigator at the Center for Gene Therapy at Nationwide, and learned about the potential treatment, the family began raising money to fund it.

That was the first glimmer of hope that I got in all of this, Cara ONeill told FoxNews.com.

A couple of months after the diagnosis in July 2013, they began a slew of traditional fundraising efforts, from bake sales and 5Ks, to parties and a golf tournament. At the end of 2013, they started the Cure Sanfilippo Foundation and have launched SavingEliza.com through the fundraising site GoFundMe.com. In April 2014, a videographer made a free video for the family that went viral, and the family started a social media campaign called Sing Two Lines, similar to the ALS Ice Bucket Challenge, where people would challenge others to sing two lines of their favorite song. Actress Andie MacDowell and members of the band Gloriana participated.

The ONeills have raised $1.7 million since December and have been able to fund the manufacturing costs of the drug as well as some of the preclinical work. Their goal is to meet the $2.3 million mark by Elizas fifth birthday, Nov. 16.

Weve made it a point not to ask previous donors to donate again, Glenn ONeill told FoxNews.com, but at some point we knew it was going to come to crunch time. At this point, with [Elizas] birthday coming up, we really need to spend more time with Eliza, and focusing on her and keeping her learning at the top of the game.

Researchers at Nationwide have proposed what they feel is a sufficient toxicology plan to move forward with the clinical trial, and they are currently waiting for the Food and Drug Administrations (FDA) comments.

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Saving Eliza: Family raising money for Sanfilippo Syndrome drug $530K away from goal