Jude doctors claim cure for 'bubble boy' disease

Infants cured of 'bubble boy' immune disease with experimental gene therapy

Gene therapy helps 'bubble boys' live in the outside world

They were born without a working germ-fighting system, every infection a threat to their lives.

As part of the study, scientists managed to alter the HIV virus so that it wouldn't cause disease - and then used it to deliver a gene that the infants lacked.

"The treatment, pioneered by St. Jude doctor Brian Sorrentino who recently died, involves removing some of the babies' blood cells, using the altered HIV to insert the gene that the patients lack - and returning the cells through an IV".

Eight infants in the United States with the condition, X-linked severe combined immunodeficiency (X-SCID), have grown into healthy two-year-old toddlers living normal lives.

It will take some years before researchers know for sure whether the therapy is effective in the long term. "He just kept getting these infections", his mom Kristin Simpson, told the AP, calling her son's condition at the time "just heartbreaking".

Now, with continual advances in treating SCID, "it's just nice to see another success for gene therapy", Kohn says. "These infants are able to respond to vaccination and are [living] normal and healthy lives". "It was made famous in the mid "70s when the 'Bubble Boy" was described in a documentary, and I think it captured the imagination of a lot of people", says Matthew Porteus, a pediatrician at Stanford University. Their findings were published Wednesday in The New England Journal of Medicine.

The 10th child is due to be released from the hospital this week, about four months after treatment.

SCID is caused by a genetic flaw that keeps the bone marrow from making effective versions of blood cells that comprise the immune system.

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The illness affects one in 200,000 newborns - nearly all males - and often causes death within the first year or two of life without treatment.

"This study marks the first time a lentivirus vector and targeted low-dose busulfan have been used as a primary treatment for newly diagnosed infants with SCID-X1", Mamcarz said.

"A simple infection like the common cold could be fatal", Mamcarz said. His life inspired the film The Boy in the Plastic Bubble, which starred John Travolta in 1976.

For years, stem-cell transplantation from a matched sibling was a patient's best hope for survival, Mamcarz said. Transplants also are medically risky - the Texas boy died after one.

Because of this, Downing has declared the therapy a "cure" for the disorder.

Previous advancements in gene therapy provided alternatives to a bone marrow transplant, but these treatments sometimes involved chemotherapy and had implications for a range of other diseases, including blood disorders, sickle cell anaemia and thalassaemia, and metabolic syndrome.

It worked by scientists creating the gene required to fix sick patients' DNA in a lab, then injecting it into their bodies inside weakened HIV viruses.

"This is the best tool we have thus far for gene therapy", said Mamcarz. The companion NIH trial evaluating the gene therapy in older children and young adults also is continuing to enroll participants.

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By contrast, in the current trial, not only did seven of eight infants develop normal numbers of functioning T cells, B cells and natural killer (NK) cells, within three to four months of receiving the therapies, but the vector used to correct the mutation was created to avoid the possibility of leukemia or any other illness developing.

The gene therapy involved collecting the babies' bone marrow and correcting the genetic defect in their DNA soon after their birth. So far, no lasting or serious effects have been observed in the babies.

Omarion is the 10th boy treated in the study, which is ongoing.

The gene therapy trial in infants is funded by the American Lebanese Syrian Associated Charities (ALSAC), and by grants from the California Institute of Regenerative Medicine and the National Heart, Lung, and Blood Institute, part of NIH, under award number HL053749.

"While longer follow-up is needed to assess any late effects of treatment, these results suggest most patients treated with this gene therapy will develop a complete durable immune response without side effects", Cowan said. "To me, this looks promising". It has an exclusive licensing agreement with Mustang Bio to develop a product.

The doctors said they have skirted the leukemia problem by implanting "insulators" around the replaced gene, preventing the treatment from accidentally activating adjacent genes and causing cancer.

"This is a first for patients with SCID-X1", she added, referring to the most common type of the disease.

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