YOUR HEALTH: Living outside a bubble

SAN FRANCISCO – A small child with a big problem.

But not on this day.

"Alright come on group hug," said a group of doctors, nurses, friends, and family who wanted to mark a milestone for eight 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 his aunt, Dannie Hawkins.

Fortunately, Dannie and Ja'Ceon found the help they needed at the University of California-San Francisco Benioff Children's Hospital.

Isolation bubbles were long gone.   Now there are 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 the University of California and St. Judes Hospital in Memphis.

"We took maybe five to ten percent of his bone marrow and we isolated the stem cells," explained Dr. Morton Cowan, professor of Pediatrics at Benioff's Children's Hospital.

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," said Dr. 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?" said Hawkins.

"hat`s why I took it cause he`s just never been out in the free world."

It's still early in the trial, but doctors say Ja'Ceon's prognosis is very promising.   The trial will treat at least fifteen children like him at the University of California-San Francisco over the next five years.

BACKGROUND: Severe Combined Immune Deficiency or SCID, is a potentially fatal primary immunodeficiency that occurs when there is a combined absence of T-lymphocyte and B-lymphocyte function. There are at least 13 different genetic defects that can cause SCID, and these defects can lead to extreme susceptibility all the way to very serious infections. This is a condition generally considered to be the most serious of the primary immunodeficiencies. Early identification of SCID can make life-saving intervention possible before infection occurs. Many states currently have SCID added to their newborn screening panel. David Vetter, affectionately known as the boy in the bubble, was born with SCID in 1971. He captured the world`s attention as he lived in protected environments to maintain relatively germ-free surroundings. This disease, as a result, is better known as the bubble boy syndrome.  (Source: https://primaryimmune.org/about-primary-immunodeficiencies/specific-disease-types/severe-combined-immune-deficiency-and-combined-immune-deficiency/)

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.com or Marjorie Bekaert Thomas at mthomas@ivanhoe.com.