UCLA doctor finds gene therapy cure for ‘Bubble Baby’ children

Eighteen children born with a tragically rare disease can look forward to healthy lives.

The children were born with the so-called Bubble Baby disease, scientifically known as severe combined immunodeficiency (SCID). Those with it have a malfunctioning immune system and must be isolated so that germs don't penetrate them.

But Dr. Donald Kohn, a stem cell researcher at UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research in Los Angeles, has developed a gene therapy that cured a group of children with adenosine deaminase-deficient SCID, a subtype of the illness that affects about 15% of SCID patients — but just 1 in every 200,000 to 1 million births around the world.

"All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems," Kohn, who spent 30 years on his cure, said in a statement.

His experiments involved removing children's blood stem cells then genetically altering them to correct their errors. The treatment was shown to have no side effects, and the next step is FDA-approval. There's hope that the approach may work in treating sickle cell disease, too.

Now, kids like Evangelina Padilla-Vaccaro, of Corona, Calif., can go to the store and get kisses from their moms.

"To finally kiss your child on the lips, to hold her, it's impossible to describe what a gift that is," her mother, Alysia Padilla-Vacarro, told ABC News. "I gave birth to my daughter, but Dr. Kohn gave my baby life."