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This technique replaces a defective version of a gene that the body needs to build cells that look for and destroy invading germs.
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Using the cunning used by the AIDS virus to infect people, doctors at two medical centers say they have cured 10 children of the so-called Bubble Boy Disease, a genetic anomaly that leaves children, usually boys, without immune system.
This technique replaces a defective version of a gene that the body needs to build cells that look for and destroy invading germs. Previous versions of the treatment were less effective and could also trigger leukemia.
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Doctors said they circumvented the problem of leukemia by implanting "insulators" around the replaced gene, thus preventing the treatment from accidentally activating adjacent genes and causing cancer.
Three months after treatment, non-defective immune cells appeared in all treated children except one. This child was successfully treated with a second cycle of gene therapy 12 months after initial treatment.
All have normal growth and development, and all the infections they have suffered because of their broken immune system have disappeared. There was no sign of leukemia.
"Children are cured because, for the first time, we are able to restore the three types of cells that make up a complete immune system," said Dr. Ewelina Mamcarz, lead author of the Bone Marrow Transplant Center. Cell therapy from the St. Jude Children's Research Hospital. in Memphis, Tennessee, said at a press conference. "Our patients are able to generate a healthy and fully functioning immune system and are now responding to vaccines, which is a first for a gene therapy trial."
The results of the first eight babies treated at St. Jude and the University of California at Benioff Children's Hospital San Francisco until September 30 are published in the New England Journal of Medicine.
These children, treated between the ages of 2 and 14 months, were followed only for 7 to 25 months; therefore further studies will be needed to determine if there are any long-term problems.
The tenth child is expected to leave the hospital this week, about four months after treatment. It usually takes three or four months for the corrected cells to sufficiently strengthen the immune system to allow a child to come out of isolation.
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Based on the results so far, said St. Jude's President, Dr. James Downing, "we are not comfortable for the moment to say it is a cure".
"Only time will tell if it is a lasting treatment that will last a lifetime," said Dr. Alain Fischer, a professor at the College de France, also a member of the University of Paris. unit of pediatric immunology, hematology and rheumatology of the Necker hospital for sick children, in Paris. He was not involved in the new treatment, but contributed to the development of an initial gene therapy for the disease 20 years ago.
This new work "represents a significant advance in the development of gene therapy and specifically for these diseases," Fischer told Reuters Health during a phone interview.
Previous treatments were less effective and less safe, although the first patient treated by the Fischer team remains alive 20 years later and that he is doing well.
Bubble Boy disease, formerly known as severe combined immunodeficiency related to X or SCID-X1, is a rare genetic defect that leaves the baby defenseless against infection. Treatment usually requires obtaining stem cells from a donor, which can be dangerous if the donor is not a brother or sister of the same size. Sister and sister donors are generally available in less than 20% of cases.
The new technique developed at St. Jude involves taking some of the baby's bone marrow and using an AIDS virus to inject a working copy of a gene called IL2RG into the cells. The gene constitutes a protein essential to the establishment of a functioning immune system. The treatment can not cause AIDS.
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Mamcarz noted that "any genetic disease with a known genetic defect lends itself to this approach".
Some previous attempts to treat bubble boy disease have only yielded temporary results. She said that this treatment does not seem to have this problem.
"In previous trials, the decline of the immune system had been observed much, much earlier – in the first year," said Mamcarz. A patient treated with the new technique has been followed for more than two and a half years, with no sign of a progressive decline in benefits.
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