In 1990, geneticist William French Anderson injected cells with altered genes into a four-year-old girl with severe immunodeficiency disorder. This was the first sanctioned test of gene therapy, in which genetic material is used to treat or prevent disease.
“If we’re lucky,” Anderson told The Chicago Tribune, “with this little girl we’ve opened the door for genetic engineering to attack major killers and cripplers, particularly AIDS, cancer and heart disease.”
Gene therapy has never fulfilled these grand hopes. In the decades since Anderson’s experiment, thousands of clinical trials of gene therapies have been carried out. But the first gene therapy was only approved for sale in the U.S. this week. The Food and Drug Administration announced its approval of Kymriah, a gene therapy produced by Novartis for a form of childhood leukemia. A few gene therapies have previously become available in China and Europe.
An FDA press release emphasizes the “historic” nature of the approval. “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb says.
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