“Bubble Boy” Disease Effectively Cured Using HIV Gene Therapy

Screengrab / NBC News / YouTube

The gene therapy used the human immunodeficiency virus (‘HIV’) to achieve remission in 10-different children.

According to NBC News, 10 babies who were born with “bubble boy disease” have been cured with HIV gene therapy. The disease meant that the babies were born without a system to fight germs. HIV, the virus that causes AIDS, cured the disease.

Scientists said they altered the HIV virus so it could not cause disease and instead would deliver a gene that the boys didn’t have.

“The children are cured," Dr. Ewelina Mamcarz, one of the study leaders at St. Jude Children's Research Hospital in Memphis told NBC News. “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."

Mamcarz said that it will take more time to make sure that the treatment is a permanent fix.

Omarion Jordan, who is nearly 1 year-old, received the treatment to treat severe combined immunodeficiency syndrome (SCID). "For a long time we didn't know what was wrong with him. He just kept getting these infections," said his mother, Kristin Simpson. Finding out that he had SCID "was just heartbreaking ... I didn't know what was going to happen to him."

Now, Omarion has a normal immune system.

The study was published by the New England Journal of Medicine. The treatment was begun by a doctor at St. Jude who recently passed away, Brian Sorrentino.

SCID, which affects 1 in 200,000 newborn babies, is caused by a flaw in the baby’s genes that keeps the bone marrow from creating effective versions of blood cells in the immune system. Without treatment, the affected babies often die in the first year or two of their lives, as a simple infection could become fatal.

The nickname “bubble boy disease” came from a case in the 1970s where a Texas boy with the disease lived in a protective plastic bubble for 12 years to remain isolated from germs. SCID can sometimes be cured by a bone marrow transplant from a sibling, but transplants are incredibly risky. The “bubble boy” died after one such transplant.

Gene therapy could solve this problem. In the treatment, some of the patient’s blood cells are removed, the modified HIV is inserted, and cells are returned through an IV. Before the cells are returned, some of the patient’s marrow is destroyed so the modified cells can grow.

In a small study of infants, normal levels of healthy immune cells developed in seven of the babies. The eighth baby needed another dose of the gene therapy but is now well. There were no lasting side effects.

Omarion is the 10th baby who received the treatment. The study, which is sponsored by the American Lebanese Syrian Associated Charities, the California Institute of Regenerative Medicine, the Assisi Foundation of Memphis and the federal government, is ongoing.

Experts are hopeful that the treatment is a cure, but more time is needed to study the treatment’s effects.

Read the full story here.

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