We think it’s too early to guess at what any therapy based on this technique would cost, so we won’t flag the story for not mentioning costs. The story says that the therapy resulted in “increased platelet counts” and “improvement of some immune-system cells.” The story should have provided quantitative data and tried to explain what these changes might mean for patients. Another problem is that the story didn’t tell us how long the patients were followed and how long the benefits lasted. The effects of some gene therapies fade with time because the cells injected with DNA by viruses ultimately die and stop producing the therapeutic protein. The story acknowledged early on and prominently that one of the study participants developed leukemia, which may have been related to the new treatment. The story probably should have mentioned that other harms might become apparent as these patients are followed further or as more patients are studied. However, we think the study went far enough to earn a satisfactory. Although the story was not grossly imbalanced and did feature some restraining quotes about the early stage of the research, we don’t think it went far enough in explaining just how preliminary this research, and the report upon which the story is based, are. Here’s where we think the story fell short: Again, while the story’s coverage was not overtly misleading, we think discussion of these issues would have put additional (and appropriate) emphasis on the need for caution when interpreting the results. The story didn’t exaggerate the consequences of Wiskott-Aldrich syndrome. The story included interviews with two independent experts who weren’t involved with the study. The story discusses one of the primary existing treatments for Wiskott-Aldrich syndrome: bone marrow transplants. The story’s muddled overview of this treatment is likely to leave readers confused, however. The story starts off its description by saying that such transplants, if they succeed, “basically cure the disease.” It then goes on to list a number of serious complications that can plague even apparently successful transplants. So the children treated via transplant sound like they probably aren’t “cured” as the story initially states. We’ll call it satisfactory, but we wish the story was a bit clearer on this point. The story never explicitly addresses the availability of this new technique or how close it may be to approval. However, it does note that the research represents just a “first step” and “proof of concept” for the new approach. Although we would like to have seen a more direct comment about the many years of testing that lie ahead for any therapy based on this technique, we think most readers will understand that this is preliminary research and not something they’re likely to have access to anytime soon. The story never mentioned that gene therapy using viral vectors is being studied for the treatment of a variety of other hereditary diseases as well as cancer. Importantly, this research has shown that it is extremely difficult to develop effective therapies based on viral vectors — context that would have been valuable in this story. This story doesn’t appear to be based on a news release.
Gene Therapy Corrected Rare Bleeding Disorder: Study