A Juneau boy with a rare genetic disease has now undergone a potentially life-saving treatment after years of fundraising.
Cade Jobsis just turned 5 years old, and with that birthday, received gene therapy treatment in Dallas, Texas – a treatment that could save his life.
Cade’s mother, Emma Jobsis, has been raising money since 2023 through a campaign she built called Cure for Cade. The goal of the funds was 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, without treatment, would gradually take away his cognitive and motor function. Cade struggles to speak and walk. He has been in intensive physical therapy since he was nine months old.
“So the hope for this drug, what the research team hopes that it does, is it stops the progression of his disease,” Emma Jobsis said. “Basically, they just kind of slowly become a shell of themselves over time. And the goal of this drug is that it stops that from happening.”
Speaking on the phone from Dallas, Jobsis said there already has been damage to Cade’s systems, but she doesn’t yet know how much can be overcome.
“In the best case scenario, this drug will bring back some of the abilities that he’s already lost, but we won’t know that for certain for a while,” she said. “And it’s not something where you just flip a switch and all of a sudden everything is the same. He still has to learn the skills that he hasn’t learned yet.”
For now, Cade is still in Texas recovering from the treatment, which gave him some expected flu-like symptoms. He’ll travel between Juneau and Texas for follow-up appointments and he’ll be on severe immunosuppressants for six months, his mom said.
The treatment has shown promising results in other children with Cade’s condition, but there isn’t a big sample size. This treatment is still in clinical trials — which were restarted with $4 million Jobsis and other families raised. She said only seven or eight kids have received the treatment, so they have very limited data. But that’s the point of the trials, she said.
“That’s what we’re doing,” she said. “Getting the information so that hopefully the next generation of kids with this disease will be able to say, ‘We have a drug and it works, and you can get it right away.’”
Jobsis said she’s grateful for Juneau residents and businesses. In just the last six months, they raised more than $1 million to close the gap left by a grant that fell through. That’s on top of previous local fundraising efforts. The money helped restart the trials so Cade and three other children with the disease could receive the treatment.
“We will never be able to repay our town for what they did,” she said. “For the chance, the hope that they gave us, and to fight with us for Cade.”
Jobsis said Cade and the family plan to return home at the end of April. She doesn’t yet know exactly what the future holds for Cade, but now, it’s his to discover.
