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Treatment for Blood Disease Is Gene Therapy Landmark


4 Star


Treatment for Blood Disease Is Gene Therapy Landmark

Our Review Summary

We were struck by how well the story was organized, structured and written.  Very easy to follow.


Why This Matters

The use of viral vectors (in this case and adenovirus) to deliver missing genes to patients with genetic disorders has been attempted for many years.  Hemophilia Type B is a genetic debilitating disorder caused by an inability to produce a protein (factor IX) that is need for blood to coagulate.  Without this critical factor, patients with Hemophilia B have spontaneous bleeding episodes.  Treatment of acute bleeding episodes consists of Factor IX infusions.  These bleeding episodes can be reduced or eliminated with routine infusion of Factor IX.  A search for an alternative strategy has been ongoing for years with viral vector treatments one obvious choice.

As one independent expert said, “After all the hype (about gene therapy) in the early 1990s….this is a terrific advance for the field.”  Definitive conclusions can’t be drawn after success in 5 of 6 patients, but this is an important step.


Does the story adequately discuss the costs of the intervention?


The story does a good job noting the possible costs of this approach ($30,000 for a single treatment) and places it in the context of current costs ($300,000 annually).  With an estimated incidence of hemophilia B at about 5 per 100,000 males, the cost of treating the disease worldwide is a important piece of the story.

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


The story states that six patients were “successfully treated” and explained that treated patients “continued to produce their own Factor IX for up to 22 months,” and notes that one patient had an initial good response but his Factor IX levels declined.  It also points out that because his immune system recognized the viral vector, it is now sensitized to respond in the future and he now can’t be treated again.  That would mean that five of six – not six of six – were successfully treated.

There was also no comment about what difference these blood test changes made in patients’ lives – how they felt.

Nonetheless, we’ll give the story the benefit of the doubt.

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


The story mentions serious problems with other delivery viruses and that liver cancer had been observed in mouse studies.  The description of the one patient’s decline – and the fact that he can’t be injected again with the same virus “because his immune system is now primed to attack it” was also important.

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


The story did a good job of putting the new findings into the context of past research and explaining the difference in this approach.

Does the story commit disease-mongering?


There was no disease mongering of hemophilia B.

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


The story quotes an editorial writer calling the trial “a landmark study.”  But that editorial writer had much more to say in that editorial – including raising specific concerns on potential harms.

The one independent expert quoted only called it a “terrific advance” but wasn’t quoted giving a specific analysis of the research – what it means or what it may not mean.

A second external voice was included – but that was a researcher who, although a competitor, was nonetheless listed as a co-author of the paper because her lab helped monitor the patients.

Does the story compare the new approach with existing alternatives?

Not Satisfactory

We think that the story could have done a better job describing the current treatments.  True, the story did note the use of Factor IX concentrate as the standard treatment but it did not describe how often the treatments are needed, the risks of those treatments (low but real risk of hepatitis and HIV) or that they work well.  The editorial writer referred to the current treatment as “cumbersome” but that wasn’t referenced in the story.

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

Not Satisfactory

It’s clear from the story that this is an experimental approach, but the story didn’t challenge a researcher’s claim that a genetic treatment for hemophilia B “could be available for widespread use in a couple of years.”  That kind of statement requires some kind of explanation of what the road to widespread use may look like…and how unpredictable such a prediction can be.

Does the story establish the true novelty of the approach?


The novelty of this approach – both for what it apparently achieved – and for how it builds on past research – was clear in the story.

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


It’s clear that this story did not rely on a news release.

Total Score: 8 of 10 Satisfactory


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