Nicholas Wade, writing in the NYT, says this in his opening paragraph about a new gene therapy for hemophilia:
Medical researchers in Britain have successfully treated six patients suffering from the blood-clotting disease known as hemophilia B by injecting them with the correct form of a defective gene, a landmark achievement in the troubled field of gene therapy. Hemophilia B, which was carried by Queen Victoria and affected most of the royal houses of Europe, is the first well-known disease to appear treatable by gene therapy, a technique with a 20-year record of almost unbroken failure [emphasis mine].
What a lie! As I’ve outlined previously, gene therapy has astonishingly high efficacy rates, 75-90% of patients in clinical trials have seen a therapeutic effect. It is a troubled field, for sure, but I would not call this a ‘record of almost unbroken failure’. Gene therapy has cured some cancers, successfully treated HIV, given sight to the blind and treated (or cured) potentially fatal immune conditions. And also has treated hemophilia, as the NYT article notes.
But of course, these successes are downplayed:
Gene therapy has had minor successes in very rare diseases but suffered a major setback in 1999 with the death of a patient in a clinical trial at the University of Pennsylvania. Another gene therapy trial treated an immune deficiency but caused cancer in some patients.
Surely if causing one death is a ‘major setback’, then saving the lives of many more patients should not be dismissed as merely a ‘minor success’.
Gene therapy, though it has proved more dangerous than was first thought, still deserves all the hype it ever received.