For years, doctors have been searching for a better way to treat children born without an immune system. The standard treatment — bone marrow transplant — comes with a risk of serious infection. Now a new treatment could be a game changer.
This marks a milestone for 8-month-old Ja'Ceon Golden.
Ja'Ceon was born with severe combined immunodeficiency; also known as the "bubble boy" disease. Without a functioning immune system, even a cold could be deadly.
Fortunately, Ja'Ceon's family found the help he needed at the University of California, San Francisco Benioff Children's Hospital. Isolation bubbles were long gone. There were two options: a bone marrow transplant or an experimental gene therapy treatment study which could lower the risk of infection.
"There's a possibility he could help himself plus other kids, then I was all for trying," said Dannie Hawkins, Ja'Ceon's aunt.
The groundbreaking effort to grow a new immune system for Ja'Ceon took a team effort between UCSF and St. Jude's Hospital in Memphis.
"We took maybe five to 10 percent of his bone marrow and we isolated the stem cells," said Dr. Morton Cowan, professor of pediatrics at UCSF Benioff Children's Hospital.
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The cells were sent to St. Jude's where researchers corrected the cells, froze them and sent them back. But before Ja'Ceon could receive the cells, he needed chemotherapy to make sure there would enough room in his bone marrow for them to grow.
"He got the chemotherapy over two days, and on the third day we infused the cells," Cowan said.
Today, Ja'Ceon can stroll down streets like anyone else.
"He's just looking around like, 'What is this and who are these people?' That's why I took it, because he's just never been out in the free world," Hawkins said.
It's still early in the trial, but doctors say Ja'Ceon's prognosis is very promising. The trial will treat at least 15 children at UCSF over the next five years that have the same x-linked version of the disease as Ja'Ceon.
SCID develops in about one in every 60,000 newborns in the U.S., of which about 25 percent have this x-linked form of the disease.
