Juneau child's clinical trial can proceed after community raises over $1 million

Juneau residents raised more than $1 million this fall to help fund gene therapy for 4‑year‑old Cade Jobsis and three other children with AP4 hereditary spastic paraplegia, allowing a paused clinical trial to restart.

A Juneau family reached a funding milestone that will allow a restarted clinical trial to move forward for a child with a rare genetic condition. Emma Jobsis has spent two years raising money after her son Cade was diagnosed in 2023 with AP4 hereditary spastic paraplegia, also called SPG50. Four families collectively raised funds to pay for drug production, and a shortfall in treatment funding led the community to step in this fall. One child in the group has already received a first dose and the others are awaiting scheduling. Key details: - Emma Jobsis and three other families raised $3 million to pay for production of the gene therapy drug. - A grant to cover treatment administration fell through in September, prompting new fundraising efforts. - Juneau residents contributed more than $400,000 in two months, and overall community giving this fall exceeded $1 million. - A local family's $600,000 donation closed the remaining gap and provided enough funding to treat all four children. - One child, Naomi, received a first dose on Dec. 9; the other three, including Cade, are waiting to be scheduled. Summary: The community fundraising effort in Juneau secured the funds needed to resume treatment in a clinical trial for four children with AP4 hereditary spastic paraplegia, and one child has already received an initial dose. Scheduling for Cade and the remaining children is expected in the coming weeks.