A Juneau boy with a rare genetic disease will be able to receive potentially life-changing gene therapy after local residents raised more than $1 million this fall.
For the past two years, 4-year-old Cade Jobsisʼs mother Emma Jobsis has been raising money to allow scientists to restart clinical trials that previously showed promising results treating AP4 Hereditary Spastic Paraplegia, or SPG50.
Cade was born with the rare disease, which would gradually take away his cognitive and motor function without treatment.
“We got his diagnosis in 2023,” Jobsis said. “And that’s when we found this group that was working on this gene therapy, but there wasn’t any funding.”
Jobsis said she and three other families who have kids with the disease started asking their communities for help. She said the Juneau community has been supportive from the start — especially the kids.
“They would knock on my door with a jar of coins, telling me they had collected them for him. And it was so heartwarming,” Jobsis said. “But it wasn’t the jar of coins that did it. It was the fact that so many kids and families were talking about him and telling people about him.”
The four families raised $3 million to pay for the production of the drugs. Then, in September, a grant that would have covered the treatment administration fell through.
The families needed another $1.15 million.
Jobsis said she was a wreck, but she didnʼt lose hope. She used TikTok, Facebook and Instagram to spread the word.
“I just got on my social media and just started talking. And boy, oh, boy, did my community hear me,” she said.
Jobsis said so many businesses in Juneau stepped up to help raise the money, she couldnʼt keep track of them all. In total, the Juneau community contributed more than $400,000 in just two months.
They were still less than halfway to their goal.
“A local family contacted me,” Jobsis said. “And she said, ‘I have been watching your social media. I have seen what you’re trying to do, and I have heard you say on countless occasions that you just need to reach the right person, and we want to be that right person for you.ʼ”
That $600,000 donation brought them across the finish line, providing enough funding to treat all four kids.
One of them, a young girl named Naomi, got her first dose of the drug on Dec. 9. Now, the other three children are waiting to be scheduled. Jobsis said they’ll find out when Cade gets his treatment in the coming weeks.
“I cannot thank this community enough,” she said. “The weather is terrible here, but we live here for the people. The community is unmatched.”