UCLA Doctors Hail Potential Cure For 'Bubble Baby' Syndrome
WESTWOOD (CBSLA.com) — Doctors say a groundbreaking stem cell therapy treatment out of UCLA may have cured "Bubble Baby" syndrome once and for all.
KNX 1070's Brian Ping reports Dr. Donald Kohn has perfected a gene therapy that has now cured 18 children born without an immune system, known as ADA-deficient severe combined immunodeficiency (SCID).
Only weeks after giving birth to fraternal twins in 2012, Alysia and Christian Padilla-Vaccaro found out their daughter Evangelina's immune system was so deficient that she could have no exposure to the outside world.
After enrolling their daughter in Dr. Donald Kohn's revolutionary stem cell gene therapy treatment - which was nearly three decades in the making - doctors extracted stem cells from the bone marrow in Evangelina's hip, then used a modified mouse virus to correct her faulty gene before replacing the marrow.
"You hear the words 'mouse virus' and you want to run the other way," said mom Alysia. "But they modify it so that it's teaching it to do something that they want it to do, which is put something in there that was missing."
Evangelina's new immune system developed without side effects and she is now living a healthy normal life.
Her mother Alysia said while the process was difficult for any mom to go through, it was all worth it.
"The reality of it is if my daughter was born the year I was born with this problem, she wouldn't be here," she said.
Doctors say the next phase of this therapy will be used to treat sickle cell disease, with clinical trials set to begin next year.