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Gene Therapy Corrected Rare Bleeding Disorder: Study


4 Star


Gene Therapy Corrected Rare Bleeding Disorder: Study

Our Review Summary

This story relayed the findings of a very preliminary study on a new treatment for Wiskott-Aldrich syndrome — a rare genetic disorder that leads to serious immune system deficiencies and problems with blood clotting. While the story did convey some caution about the early stage of this research and the potential for major side effects, it didn’t provide quite enough detail to satisfy many of our criteria. A more thorough investigation would have emphasized just how far this technology has to go before it can be widely adopted.


Why This Matters

Because of its devastating effects on the immune system, Wiskott-Aldrich syndrome puts sufferers at increased risk of death from infections and cancer. Although bone marrow transplants can be an effective and durable treatment, many children have problems finding a good match. In addition, bone marrow transplants come with a host of serious risks and are not a treatment option for everyone. New treatments, such as the experimental gene therapy discussed in this story, are badly needed.


Does the story adequately discuss the costs of the intervention?

Not Applicable

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.

Does the story adequately quantify the benefits of the treatment/test/product/procedure?

Not Satisfactory

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.

Does the story adequately explain/quantify the harms of the intervention?


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.

Does the story seem to grasp the quality of the evidence?

Not 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:

  • There was no specific acknowledgment of the limitations of study that involves only 10 people. Although savvy readers may recognize the drawbacks, others many may not understand that the results cannot necessarily be applied to the broader population of patients with this condition.
  • There was no mention of the fact that the study did not include a control group for comparison. Results of uncontrolled studies typically appear more favorable than those which include a control group.
  • The story did not include a caveat about the limited peer review that conference papers typically are subjected to.

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.

Does the story commit disease-mongering?


The story didn’t exaggerate the consequences of Wiskott-Aldrich syndrome.

Does the story use independent sources and identify conflicts of interest?


The story included interviews with two independent experts who weren’t involved with the study.

Does the story compare the new approach with existing alternatives?


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.

Does the story establish the availability of the treatment/test/product/procedure?


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.

Does the story establish the true novelty of the approach?

Not Satisfactory

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.

Does the story appear to rely solely or largely on a news release?


This story doesn’t appear to be based on a news release.

Total Score: 6 of 9 Satisfactory


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