Cure Sanfilippo Campaigns Raise $4M to Date, Support Therapy Trials

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by Mary Chapman |

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Cure Sanfilippo Foundation campaigns

The Cure Sanfilippo Foundation is on track, with more than $1 million raised in a GoFundMe campaign, to support a potential gene therapy trial for Sanfilippo type C.

This brings the total to date to about $4 million collected through three separate, ongoing campaigns helping to fund clinical studies of potential therapies.

While each effort centers on a family’s particular story, they are all imbued with the same hope: that their child will have a bright future. Sanfilippo, a genetic disorder that leads to a form of childhood dementia, is thought to affect between 1 in 50,000 and 1 in 250,000 individuals globally. Each campaign is accompanied by a three-minute video.

The first, “Saving Eliza,” went viral in 2014 and is said to have become the first campaign to raise $1 million, and then $2 million, through GoFundMe, billed as the world’s largest crowdfunding platform.

It features the O’Neill family’s battle to fund a clinical study that would give young Eliza a chance at life. Money raised — the campaign is ongoing — helped make possible a gene therapy trial for Sanfilippo types A and B. Through the trial, which is still underway, some 30 children have received treatment. (The nonprofit’s release did not specify the trial being supported, and several gene therapy trials in Sanfilippo syndrome are underway.)

“In 2014, we were just parents desperate to save our daughter, and GoFundMe provided us an outlet for sharing our story and empowering people with a way to help,” Glenn O’Neill, Eliza’s father and a Cure Sanfilippo Foundation co-founder, said in a press release.

“We hoped funding the clinical trial would be possible. And because of thousands and thousands of kind people who heard our story, donated to help, and shared our need with others, we succeeded and were able to help fund the clinical trial for many children,” O’Neill added.

“We continue partnering with GoFundMe because it provides an easy and personalized way for the foundation and families to share their stories in order to accelerate research to cure Sanfilippo syndrome.”

In recognition of the Saving Eliza campaign’s impact, GoFundMe dedicated its board room to it.

The second campaign, launched three years ago, was the Sakar family’s “Saving Carter,” an effort that has pulled in more than $1 million, thanks in part to support from celebrities such as Oprah Winfrey, Katie Perry, Mark Wahlberg, Selena Gomez, and Madonna. The funds led to an ongoing clinical study that will gauge whether lowering inflammation levels in patients might enhance their life quality. The trial, also not identified, is treating 20 children of various ages and Sanfilippo types.

The latest campaign, called “Save Connor,” opened in 2020 and focuses on Connor Dobbyn and his parents’ effort to raise $3 million for a first gene therapy trial for Sanfilippo type C, a rare subtype of this rare disease. To date, the campaign has surpassed its initial $1 million goal, with $1,050,060 raised for the trial, for which preclinical work has been conducted.

This gene therapy seeks to deliver a healthy copy of the HGSNAT gene, supplanting the defective one. That involves inserting the corrected gene into a small circular piece of DNA called a plasmid, which would be delivered to the brain by an adeno-associated virus (AAV) engineered to be harmless.

Last month, the foundation shared the story of a group of young Connor’s closest friends — “Connor’s Crew” — who have been raising funds on their buddy’s behalf.