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Gene therapy cures ALD 5.11.09 Two 7-year-old boys with a fatal brain disease called ALD (adrenoleukodystrophy) who couldn’t get bone marrow transplants were saved by scientists whose gene therapy technique may let doctors treat other incurable disorders. Doctors in Paris delivered the gene into the boys’ bodies using HIV, the virus that causes AIDS. The virus, stripped of genetic material that makes it toxic, integrates permanently into the DNA of cells it enters, scientists said. That means the modified gene remains in the blood-forming stem cells for the life of the patient, according to a report in the journal Science. Two years after the experimental treatment, the neural damage has been halted or reversed and the two boys attend school and lead normal lives, said Nathalie Cartier, the study’s lead author. The treatment was cited as an example of a “comeback for gene therapy,” after years of setbacks, in an editorial in the journal. “Their disease is completely stabilized, they are fine and there’s no reason this should change,” Cartier, research director at the French National Institute for Health and Medical Research in Paris, said in a telephone interview yesterday. If more studies confirm the results, she said, “We think gene therapy could become a first-line treatment. It’s definitely a strategy that could be applied to other conditions.” Today’s report “represents a long-sought rewarding achievement in the field of gene therapy,” wrote Luigi Naldini, a researcher in Milan, Italy, in the editorial accompanying the study. |
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