The concept behind gene therapy has always had great appeal. You have a disease caused by a bad gene? Install a good version of the gene and it might well compensate. Nothing succeeds in medicine like a quick fix. If it works.
Of course, it turned out that genes work in far more complex ways than once imagined. And there’s the pesky little matter of putting the genes into the cells where they are needed. Oh, and not killing your patient.
As Nicholas Wade writes in Saturday’s New York Times, gene therapy is “a technique with a 20-year record of almost unbroken failure.” But, in the same story, Wade reports that British gene therapists have “successfully” treated six patients with hemophilia B, which afflicts about 20 percent of hemophiliacs. He cites figures in a commentary in the New England Journal of Medicine, to the effect that a single $30,000 gene treatment may replace a cost of $300,000 a year over a lifetime for repeated doses of clotting factors.
A close read of Wade’s piece reveals that only four of the six patients were better enough to forego conventional treatment entirely. The other two got some benefit but still needed injections of clotting factor. He also doesn’t help readers recognize that six patients is a very small study though, of course, when you get a dramatic result in even a few patients, that’s pretty intriguing.
Deena Beasley‘s story for Reuters devotes a good bit of space to the economic consequences not for patients or the health care system but for manufacturers of the clotting factors. If this thing works, it could be bad for business.
The AP‘s story, by Mike Stobbe, took care in the second graf to say the study was “preliminary” and involved “only six patients” and that earlier promising attempts with gene therapy “ultimately failed.” Excellent context. Unlike most other accounts, Stobbe also gave the actual results: Patients saw their clotting factor levels increase from less than 1 percent of normal to at least 2 percent and, in one case, to 11 percent. Good tempering of irrational exuberance. USA Today picked up Stobbe’s story.
-Boyce Rensberger
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