How a new treatment could offer a way to live "outside the bubble"

Margot Kim Image
Sunday, October 22, 2017
How a new treatment could offer a way to live "outside the bubble"
They are looking today at two options: a bone marrow transplant, or an experimental gene therapy treatment study which could lower the risk of infection.

FRESNO, Calif. (KFSN) -- Children born without an immune system, traditionally have an agonizing wait for a bone marrow transplant.

"Alright come on group hug. Thank you for everything,"

Simple human contact is 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.

"I'm thinking he's going to have to live in a bubble," said Ja'ceon's aunt, Dannie Hawkins.

Fortunately, Dannie and Ja'ceon found the help they needed, isolation bubbles are long gone.

They are looking today at 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 his self plus other kids then I was all for trying," said Hawkins.

The groundbreaking effort to grow a new immune system for Ja'ceon took a team effort between two medical centers, UCSF and Saint Jude's.

"We took maybe five to ten percent of his bone marrow and we isolated the stem cells," said Morton Cowan.

The cells were sent to Saint 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," said Cowan.

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 cause he's just never been out in the free world," said Hawkins