Gene Therapy Saves Immune-Deficient Children
April 27, 2000 -- By transfusing them with a corrected gene, French physicians have restored the immune systems in five children whose rare disorder had forced them to live in a virtual "bubble" because they couldn't fight off even minor infections.
While it is too soon to say the children are cured, exultant researchers around the globe are calling this the first real proof that gene therapy works.
"The children have recovered completely normal immunological function. They are at perfect curves for development, body weight, and height. From a physical and biological point of view, they are completely normal," Marina Cavazzana-Calvo, MD, the lead researcher on the gene therapy team, tells WebMD. "This is real medical progress." She spoke to WebMD from a hospital in Paris where the children received care.
Even researchers in the U.S. are popping champagne corks. "This is the most successful of all early achievements in gene therapy," says Jennifer M. Puck, MD. "Everyone feels like they had a part in this, and we are all very pleased they were successful. This is really an international achievement." Puck directs gene therapy research for immune disorders for the National Institutes of Health. Her team identified the genes responsible for this disease and developed the gene transfer system that was used by the French physicians.
"No one has done successful gene therapy before in humans -- anywhere. There were some attempts for certain [heart] diseases, but there have not been any successes of this magnitude," says Rebecca H. Buckley, MD, PhD, the director of Duke University's bone marrow program for people with the disorder exhibited by the French babies. The program is the largest of its kind in the nation.
Although it is rare, many people have heard of this disorder, called severe combined immunodeficiency disease, or SCID, thanks to a movie about a child dubbed "the bubble boy" because he had to live in an enclosed environment. People with SCID lack a gene that allows their blood cells to produce cells that fight disease. People with AIDS have low numbers of T-cells, one of the type of fighter cells that SCID patients also lack.
Gene Therapy Frees 'Bubble Children'
April 27, 2000 -- By transfusing them with a corrected gene, French physicians have restored the immune systems in five children whose rare disorder had forced them to live in a virtual "bubble" because they couldn't fight off even minor infections.
While it is too soon to say the children are cured, exultant researchers around the globe are calling this the first real proof that gene therapy works.
"The children have recovered completely normal immunological function. They are at perfect curves for development, body weight, and height. From a physical and biological point of view, they are completely normal," Marina Cavazzana-Calvo, MD, the lead researcher on the gene therapy team, tells WebMD. "This is real medical progress." She spoke to WebMD from a hospital in Paris where the children received care.
Even researchers in the U.S. are popping champagne corks. "This is the most successful of all early achievements in gene therapy," says Jennifer M. Puck, MD. "Everyone feels like they had a part in this, and we are all very pleased they were successful. This is really an international achievement." Puck directs gene therapy research for immune disorders for the National Institutes of Health. Her team identified the genes responsible for this disease and developed the gene transfer system that was used by the French physicians.
"No one has done successful gene therapy before in humans -- anywhere. There were some attempts for certain [heart] diseases, but there have not been any successes of this magnitude," says Rebecca H. Buckley, MD, PhD, the director of Duke University's bone marrow program for people with the disorder exhibited by the French babies. The program is the largest of its kind in the nation.
Although it is rare, many people have heard of this disorder, called severe combined immunodeficiency disease, or SCID, thanks to a movie about a child dubbed "the bubble boy" because he had to live in an enclosed environment. People with SCID lack a gene that allows their blood cells to produce cells that fight disease. People with AIDS have low numbers of T-cells, one of the type of fighter cells that SCID patients also lack.