Cure for Cade raises enough to continue with clinical trials for child’s rare genetic disease
- Ellie Ruel
- 7 hours ago
- 3 min read
Weeks of fundraising gets $1.15M needed for hospital treatment in addition to $3M already raised for special drug
By Ellie Ruel
Juneau Independent
After months of fundraising, the Jobsis family has reached their $1.15 million goal to enter 4-year-old Cade Jobsis in clinical trials to treat his rare genetic disease.
“I keep saying I feel a little numb right now because I honestly wasn't sure we would ever make it happen, and especially not as fast as we did,” said his mother Emma Jobsis.
She had been fundraising for six weeks to keep trials on track to begin at the end of this year. About two months ago, the family learned that an additional $1.15 million beyond the $3 million they already raised with three other families for the cost of the drug itself would be needed to pay for hospital treatment.
In the last couple of months, Cure For Cade has received support through GoFundMe donations. Multiple local businesses hosted fundraisers, like a September silent auction, raffle and bake sale at Tracy’s King Crab Shack, along with more than 15 concurrent efforts by other establishments. More recently, a Halloween haunted garage collected Cure for Cade donations as an admission fee.
“We couldn't leave our house without somebody stopping us and talking to us about the fundraising,” Jobsis said. “And I think one of the most powerful things about this, this fundraiser, is it was something that brought everyone together. Everyone supported this, and everyone rallied around this, and it was just really incredible to see a community come together.”
Cade was diagnosed with hereditary spastic paraplegia, SPG50, two years ago. It’s a rare neurodegenerative disorder that can lead to loss of cognitive and motor function and eventually more serious complications like epilepsy.
“Cade, he received a bad copy from me and a bad copy from my husband of the exact same gene,” Jobsis said. “Gene therapy, the idea is that it'll give him back that copy, that gene that he's missing, basically an instruction manual that his body doesn't have to produce that protein.”
The therapy was originally pursued by Terry Pirovolakis, whose son was diagnosed with SPG50 at 15 months old. Pirovolakis partnered with biomedical researchers to develop the gene therapy Melpida, the name of which is a combination of his son’s name, Michael, and the Greek word for hope, Elpida.
Jobsis said that the rarity of SPG50 makes finding research and funding difficult overall. There’s only about 90 reported cases of the disease worldwide, according to National Institute of Health data from 2023.
“Other parents like us were reaching out to him, saying, ‘How did you do this? How did you get this?’ And we were really like, ‘We need this to be available to families, not just a one-off, compassionate use dose, but available clinically,’” Emma Jobsis said. “There's a huge gap in medicine and research for rare and ultra-rare diseases. There just isn't, there isn't research. There aren't any clinical trials there. There isn't because there's so few patients that nobody's willing to fund it, which is why we're doing it.”
The first child in the trial cohort is set to begin treatment in Texas in early December. The families will know who the next participant will be by the end of 2025.
In the meantime, Jobsis said it’s a waiting game. Cade goes to preschool four days a week and physical therapy after that.
“The biggest blessing for us is that Cade doesn't know anything's wrong. He doesn't know that his body is failing him. And that is such a blessing to us in this process, because if he did, that would be awful,” she said. “It's really hard to know that that could all be taken away really soon if we don't get him this gene therapy.”
Jobsis said she’s grateful for the outpouring of support they’ve seen.
“Thank you to our community for standing by us with this,” Jobsis said. “It really, I think, showcased what a special place this is, and while the weather can be hard living here, I don't think we could ever live anywhere else because of how special the people are and how much we support each other.”
• Contact Ellie Ruel at ellie.ruel@juneauindependent.com.
