Clinical trial helps Valley toddler overcome 'bubble boy' diseasePosted: Updated:
A new gene therapy could change the lives of children that have the so called "bubble boy" disease.
For the last 11 years, stem cell researchers have been testing 10 children with the disease.
As part of the study, doctors remove bone marrow from a child, add a normal copy of the deficient gene in the lab and then transplant it back into the child.
An Arizona family joined the trial in 2008.
CBS 5 talked with Colton Ainslie, an energetic 5-year-old. His life, though, didn't start out that way.
When Colton was just a few days old, his parents, Michael and Jessica Ainslie, knew something was wrong.
"He started getting really bad cough, vomiting and throwing up. He got thrush - all the white stuff in the back of the throat. He got that really bad," said Jessica.
After multiple tests, Jessica and Michael found out their son had severe combined immune deficiency, also known as SCID or "bubble boy" disease.
Colton's immune system had a difficult time fighting off infections so he had to stay at home, isolated from possible germs.
"It was hard because we want to go to the store and do normal things, but we can't because we can't take our kids with us," said Colton's mother.
So Colton's parents decided to join the clinical trial of a gene therapy regimen out of UCLA, in hopes of restoring his immune system.
"We took him into surgery, they took out his bone marrow. They gave him two doses of chemo - a light dose of chemo. Wiped his cells out completely so everything was zero they gave him his own bone marrow back. They modified it," said Jessica.
So far, the therapy has worked.
Colton's little sister, Abbygail, also has the disease. But she is also doing well with the gene therapy.
Michael and Jessica now hope this is the beginning of a life beyond the walls of their home.
Michael said he just wants his children to live "normal, happy, healthy lives."
Jessica said that Colton talks about being an astronaut, "If you want to be a SCID patient and you're the first man that has SCID and goes on the moon - go for it. I want him to have hope."
"I just don't want my kid to be in a hospital bed for the rest of their life. I want them to be who they are - what God made him to be," said Jessica.
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