​a Groundbreaking Cure for ‘Bubble Babies’

ByAnisa Arsenault
Associate Editor
Published
Nov 2014
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“To finally kiss your child on the lips, to hold her, it’s impossible to describe what a gift that is,” says mom Alysia Padilla-Vacarro. For the first months of her daughter Evangelina’s life, hugs and kisses just weren’t possible. That’s because Evangelina is one of 100,000 babies born each year with a malfunctioning immune system, called  Bubble Baby disease. But now, she’s one of 18 infants who’ve been cured.

The disease, scientifically known as severe combined immunodeficiency (SCID), strikes a baby’s immune system and requires them to be isolated to avoid contact with germs. Evangelina was among a smaller subset of SCID babies who had ADA-deficient SCID, meaning their cells didn’t create the white blood cell-building enzyme ADA. But thanks to  Dr. Donald Kohn, a stem cell researcher at UCLA, 18 babies born with ADA-deficient SCID are going to live healthy, normal lives.

"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,” says Dr. Kohn.

After thirty years, he perfected what seems to be a risk-free form of stem cell gene therapy, in which the disease-causing faulty gene is corrected using the baby’s own cells. The procedure involves removing a baby’s blood stem cells from bone marrow and genetically altering them, then re-introducing them back  in the bone marrow. No more lifelong weekly enzyme injections; the stem cells regenerate and repopulate the blood stream, creating a healthy immune system.

What’s even more promising: this new form of stem cell gene therapy can potentially help other childhood diseases beyond Bubble Baby. In 2015, clinical trials will begin to see if this gene therapy can treat sickle cell disease.

 

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