Close Ad

Babies With "Bubble Boy" Disease Get New Lease on Life Thanks To Amazing Cure
Baby hospital
Physical Health

Babies With "Bubble Boy" Disease Get New Lease on Life Thanks To Amazing Cure

There’s nothing more devastating than the loss of a child. For far too many children, the rare genetic disease called severe combined immunodeficiency syndrome, or SCID, has been a death sentence – often within the first two years of life.

Until now, when some hope has been found via the amazing doctors at St. Jude’s, and the cure that came from a most unexpected place.


"The children are cured," said Ewelina Mamcarz, one of the doctors who led the study at St. Jude Children's Research Hospital. "They came to us as little infants, some of them as young as 2 months, with severe infections. Now they are home, living normal lives, attending daycare."

While the babies won't need monthly injections, it remains to be seen if the cure is truly permanent.

Omarion Jordan, who turns 1 later this month, One of the babies who was treated by the gene therapy in December is Omarion Jordan. The one-year-old was injected with a virus that delivered a gene he lacked, which was causing severe combined immunodeficiency syndrome, or SCID.

"For a long time we didn't know what was wrong with him. He just kept getting these infections." Omarion's mom, Kristin Simpson said it "was just heartbreaking ... I didn't know what was going to happen to him."

As a result of his gene therapy, Omarion has a normal immune system now. "He's like a normal, healthy baby," said Simpson. "I think it's amazing."

The results of the study on the treatment -- which was pioneered by the late Dr. Brian Sorrentino -- were recently published in the New England Journal of Medicine.

Remembering her colleague, Dr. Mamcarz told NBC News: "This trial is his life's work."

SCID prevents bone marrow from making functional blood cells, which make up the immune system. The syndrome is seen in roughly 1 in 200,000 newborns, almost exclusively male. Left untreated, SCID is usually fatal within the first few years of life. Without a functional immune system, loss of life can be caused by something as common as cold.

The syndrome first became known as “bubble boy” disease in the 1970s, when a boy afflicted with SCID lived 12 years in a protective plastic bubble to shield him from the world. He then got a bone marrow transplant, which can cure the disease if successful, but is very risky. Unfortunately, the first "bubble boy" lost his life.

When the doctors decided to try gene therapy, it was risky too. Past attempts led to children developing leukemia later, if they survived. This time, however, normal levels of healthy immune system cells developed in eight baby boys.

"So far it really looks good," said Dr. Anthony Fauci, head of the National Institute of Allergy and Infectious Diseases, which assisted with the development of the gene treatment.

As research and trials continues on this cure, and as the former "bubble boys" continue to be monitored for any further health issues, doctors hope its concept can eventually spread to more common diseases, like sickle cell disease.

Here's hoping for more (not so) little miracles thanks to the hard work and ingenuity of the medical community.

Hot Stories

Why Jennifer Hudson Forgave Her Mother's Killer -- Life Stories By Goalcast

At the peak of her career, Jennifer Hudson faced a horrific family tragedy. Hudson's mother and brother were brutally attacked in their family home, and her seven-year-old nephew was kidnapped. What followed was a heart-wrenching manhunt to rescue her nephew and seek justice against the man who took her family. Hudson speaks from the heart in this video on what happened to her family and how she was able to move forward.

Celebrities
ideas and thinking

bright ideas

The 21st century has brought great new opportunities for entrepreneurs. The digital revolution has completely changed the way we live -- and the landscape for aspiring CEOs and innovators.

powerful-business-woman

Keep ReadingShow less
Entrepreneurs
ideas and thinking

bright ideas

The 21st century has brought great new opportunities for entrepreneurs. The digital revolution has completely changed the way we live -- and the landscape for aspiring CEOs and innovators.

powerful-business-woman

Keep ReadingShow less
Entrepreneurs