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Gene Therapy Appears Successful For Two Immune-Deficient Infants

By Rick Weiss
THE WASHINGTON POST -- Two infants born with a life-threatening immune system disorder that had forced them to live inside protective sterile “bubbles” are healthy and living normal lives almost a year after being treated with an experimental genetic therapy, doctors reported Thursday.

If the children retain their good health, they will go down in medical history as the first to be definitively cured by gene therapy, a controversial approach that seeks to treat diseases by giving people new genes.

“They have complete restoration of immune system function,” said Marina Cavazzana-Calvo of Necker Hospital in Paris, who led the research with co-worker Alain Fischer. “The word “cure’ is hard to use because we don’t know how long these results will last. But the follow-up of one year is very encouraging.”

Cavazzana-Calvo said the team has recently treated three additional infants, including one from the United States, and at least two of them appear to be completely healthy. The children’s identities are not being released, to protect their privacy.

Experts in this country said they could hardly contain their excitement. “This is something you dream about,” said William Shearer, chief of immunology at Texas Children’s Hospital in Houston. Shearer, also of the Baylor College of Medicine in Houston, was one of the doctors who in the 1970s and 1980s cared for David the “Bubble Boy,” who suffered from the same disease as the French children and died in 1984 at the age of 12.

The French results, Shearer said, “are fabulous.”

In the 10 years since a 4-year-old Ohio girl with a related immune system disorder became the first person to be treated with gene therapy, doctors have tried to deliver curative genes to thousands of patients with many different diseases, all to no avail.

In 1995, a federally appointed panel criticized scientists for experimenting on too many people before enough basic research had been done.