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Doctor Uses Stem Cells to Cure 'Bubble Baby' Disease

Evangelina Vaccaro/CBS News

The Short of It

A revolutionary new treatment that uses stem cells offers a cure for children diagnosed with a severe immune disease sometimes called "bubble baby" syndrome.

The Lowdown

Alysia Vaccaro says she knew something was wrong with one of her twin daughters, Evangelina, because she was able to compare her to her sister. It turns out her mother's intuition was right. Evangelina had a genetic condition, Severe Combined Immunodeficiency, or SCID, which affects the immune system so that it can't fight off infection. Even a simple cold can be deadly. According to the National Institutes of Health, SCID affects at least 1 in 50,000 to 100,000 newborns, who are born without functioning lymphocytes that protect the body against bacteria.

"We wore masks; we had hand sanitizer; we had raw hands from cleaning so much," Vaccaro told CBS News.

Then they enrolled Evangelina in a clinical trial with Dr. Donald Kohn at the Broad Stem Cell Research Center at UCLA. Kohn took bone marrow from the baby and gathered stem cells. He then cloned a gene to replace what Evangelina was missing at birth.

"Those stem cells are given back to the patient where they can go back to the bone marrow and make the blood cells for the rest of the patient's life," Dr. Kohn explained.

The Upshot

Incredibly, 23 patients, including Evangelina, have achieved restored immune systems since being enrolled in the trial. Now 3 years old, she is in perfect health according to doctors.

"It is a cure. I know it's a cure. We're living the cure," beams the little girl's mom.

Dr. Kohn hopes to make the SCID treatment available nationwide. He's also testing the same method as a cure for sickle cell disease. Clinical trials for that treatment are now underway.

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