[L]ast year, [Jay] Konduros enrolled in a clinical trial, receiving an experimental gene therapy at Children’s Hospital of Philadelphia [to treat his dangerous blood disorder, hemophilia B]. Almost immediately, he began producing the missing clotting factor.
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And its effects lasted; after a year and a half, the longest patient follow-up, the therapy was continuing to work. The results, published [December 6] online in the New England Journal of Medicine, represent “another example of the gene-therapy renaissance,” Matthew Porteus, a pediatrician at Stanford University, wrote in an accompanying editorial. He said the data suggests that the treatment ultimately might provide an “ideal cure” for hemophilia B.
Still, he noted, the study has some limitations. The follow-up period was relatively short, from 28 to 78 weeks in the new report; longer-term studies are needed to prove safety and effectiveness over extended periods. In addition, he said, researchers need to find ways to provide the therapy to a broader group of people, including children, and to figure out how to reduce the costs involved.
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“People were planning their lives around hemophilia, and now they are doing activities that they weren’t before,” [researcher Lindsey] George said. “One man who came in a wheelchair is now out of a wheelchair and is coaching Little League.”
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