A small, early-stage study published in the New England Journal of Medicine found that a single intravenous infusion of a novel bioengineered gene therapy treatment helped spare nine of 10 hemophilia B sufferers from repeated injections of blood-clotting factor to prevent debilitating bleeding episodes.
This well-written story shares that information carefully and clearly. However, more details about the years-long road ahead for this treatment’s development and about possible ties between the researchers and the funders would have made the piece stronger.
Complicating the care taken by the reporter, however, is a glaring headline that promises much more than this small study can deliver (“Gene therapy makes a big advance treating hemophilia B blood disorder”). Whether this particular treatment revolutionizes the landscape for hemophilia B sufferers will remain an open question for some years.
People who have inherited the hemophilia B bleeding disorder face a lifetime of replacement therapy: injecting clotting factor when life deals them a stumble or a scrape. Gene therapy research appears to be on the cusp of offering an alternate treatment that would allow these individuals to lead normal lives. While it is important for journalists to track progress toward such an important goal, it is also critical that stories tread cautiously to avoid giving false hope at early stages.
Although this therapy is not yet available for patient use, the story makes a point of noting that the cost may well be “stratospheric,” should it make it to market.
If approved, such treatments are expected to command stratospheric prices. For example, a separate Spark gene therapy for a rare type of childhood blindness, which is on track for Food and Drug Administration clearance soon, could cost $1 million per patient for infusions in both eyes, according to some Wall Street analysts.
Still, the therapies can produce at least some offsetting savings. Porteus noted in his editorial that the hemophilia therapy resulted in savings of about $200,000 a year per patient because of the elimination of clotting-factor infusions.
The story summarizes the outcome of the small, 10-person trial that dominates this narrative:
Lindsey George, the lead investigator and a hematologist at Children’s Hospital of Philadelphia, said that nine of the 10 men in the trial didn’t experience any bleeding episodes after the gene therapy treatment. The one patient who needed clotting factor because of bleeding used 91 percent less than before, she said.
Although the story does not address harms directly–and should have–it does note that one of the worrying side effects of gene therapy—spurring the body to attack itself—was not present in this study.
It is clear from the story that the study is small and an “early-stage trial.” One source also notes that the researchers tracked individuals in the study for no more than a year and a half, making longer term assessments unavailable. The story could have done more, though, to signal to the reader that, despite the encouraging outcomes of the study, the treatment is still very much under development. The next stage—Phase 3 trials—requires more participants, more careful study design, and takes, on average, three years to complete.
The treatment described here, if ultimately proved worthy, could have a profound impact on a serious genetic condition.
Independent sources are identified in the story, as are the funders. But missing from the text is information describing linkages between the two funders—Spark Therapeutics and Pfizer—and the lead researchers. It’s not clear from the story that there is a financial relationship.
Individuals coping with hemophilia B have few options, and the story describes the main one: injections of blood-clotting factor to stem runaway bleeding.
Since the story notes that the research is at an early stage with only 10 participants, a reader can easily infer that the treatment is not yet available. However, additional information about remaining evaluation stages for this treatment would have been a useful reinforcing addition. Hemophilia B sufferers who see this story will likely be contacting their doctors.
The story establishes novelty with this:
The researchers presented earlier findings on the gene therapy a year ago at the American Society of Hematology’s annual meeting, but the latest data covers a larger patient group and a longer time period.
A news release issued by Children’s Hospital of Philadelphia did a good job of describing this study; the Post story could have picked up more of the salient details from that text but reveals independent reporting nevertheless by including sources not connected to the study.