Finding the cure for Severe Combined Immunodeficiency Disease

Finding the cure for Severe Combined Immunodeficiency Disease

Three years ago, little Evangelina's parents had to make sure their little girl didn't even catch a cold, as it could have killed her. The little girl had "bubble baby" disease. Read Evangelina's story and how she was cured, in this article.

Three years ago and just a few days after bringing home her twin baby girls, Alysia Vaccaro’s mummy intuition told her that little Evangelina wasn’t as healthy as her sister.

And she was right. Evangeina has been born with Severe Combined Immunodeficiency Disease (SCID), also known as “bubble baby” disease.

SCID is a genetic disorder that causes the body to have an extremely weakened immune system. Babies with this disease are extremely vulnerable to infections — even the common cold could have deadly repercussions.

A CBS News report quotes the American Academy of Allergy, Asthma and Immunology as saying that babies with SCID “often come down with recurrent, severe respiratory infections that can be life threatening.”

Alysia tells the news agency, “We wore masks, we had hand sanitizer, we had raw hands from cleaning so much.”

bubble baby disease cure

Evangeline when she was born (left) and now, a healthy 3 year old.

Looking for, and finding a solution

It’s not hard to imagine the stress the Vaccaros were under with having to ensure that not one germ entered their little girl’s system.

Looking for a solution, they turned to Dr. Donald Kohn at the Broad Stem Cell Research Center at UCLA, and enrolled their little girl in a clinical trial he was conducting there. Dr. Kohn’s specialty is developing new methods for the treatment of genetic diseases of blood cells.

And it looked like they found a solution to their daughter’s problem. Evangelina is now three years old and the picture of health.

The cure?

Dr. Kohn’s treatment “involves taking bone marrow from the patient to gather stem cells. A cloned gene is then added to correct what was missing at birth,” say reports.

These stem cells are then given back to the patient, where they enter the bone marrow and make the blood cells for the duration of the patient’s life.

Up to now the treatment has corrected the immune systems of all 23 patients who participated in the clinical trials, including little Evangelina.

In cases like this, Science certainly brings us hope, right parents?

If you could wish for a cure for one disease that affects children, what would it be? Tell us in a comment below. 

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