This story is one of two that we are reviewing (the other is from the New York Times) that focuses on a recent paper published in the New England Journal of Medicine. At issue is a novel gene therapy used to limit or eliminate symptoms related to the disease adrenoleukodystrophy (ALD).
The AP story does a good job of making clear that the treatment is not yet available for widespread clinical use and does a fair job of describing the study and its results. However, the story appears to mischaracterize current treatment options for the disease, and overlooked the potential harms.
Left untreated, ALD often leads to death within 1-10 years of diagnosis. And there are precious few treatment options available. What’s more, the primary treatment, bone marrow transplantation, relies on the availability of a donor — and poses significant health risks of its own. All this makes new treatment options incredibly important for ALD patients and their loved ones. However, the fact that we are talking about the life and well-being of patients — usually children — also makes it incredibly important to place the work in context. Limitations, availability and unanswered questions are always key points worth making, and especially so when many parents are eager for information about something that may affect the health and well-being of their children. The story does a decent job here. But the story also appears to provide an unnecessarily limited view of potential treatment options. ALD is already a daunting diagnosis with limited treatment options available. Characterizing those options as being even more limited than they already are is problematic.
The story does not discuss cost, but it does at least acknowledge that cost may be an issue. As the story notes: “The main questions are how long the benefits will last, how much the treatment will cost if it wins approval, and whether it will replace stem cell transplants from donors, he wrote” (emphasis added). The New York Times was stronger on this point, disclosing the risk that costs might be “absolutely crazy.”
The story notes that, two years after treatment, 15 of the 17 patients who received the gene therapy treatment for ALD “were neurologically stable.” That’s sufficient to earn a satisfactory rating here. However, it would have been better if it had clarified what “stable” means in this context. Did patients not display any additional symptoms of ALD after the beginning of treatment? Did the condition worsen and then improve? Or did the condition plateau at a certain point after treatment began?
Potential adverse effects were not addressed at all. According to a supplement of the journal article, side effects ranged from fever and seizures to gastroenteritis. The side effects listed in the supplement also included those for one patient (2018), whose ALD symptoms worsened significantly after treatment began. The story does note that one of the 17 patients did die from a worsening of the disease. It’s not clear what effect, if any, the treatment had on the relevant patient. However, the story does not note the side effects experienced by any of the 15 patients who became neurologically stable. Would most parents accept these side effects as acceptable risks, given the severity of ALD? Probably. But they need to be mentioned.
The release does a fair job of describing the study. It’s worth noting that this story also makes clear that it remains unclear how long the relevant benefits observed here may last. That’s an important point, and one that’s also missing from the New York Times story.
The story discloses that Bluebird Bio, the company that hopes to market this gene therapy, paid for the study. The story also notes that “some researchers work or consult for the company.” That’s enough to earn it the Satisfactory rating.
However, the story also quotes one of those researchers directly, but does not note that this specific researcher (David Williams) has conflicts of interest. According to an appendix of the journal article, Bluebird Bio also funds other projects by Williams. The company has also licensed one of Williams’ patents. That doesn’t necessarily mean that anything in the study is shady, but conflicts of interest should be acknowledged — and this story would have been stronger if it had made note of Williams’ conflicts of interest specifically.
The story states that “The only effective treatment [for ADL] is a stem cell transplant from a matched sibling.” That’s not accurate. Bone marrow transplants have to come from matched donors, but those donors are not necessarily siblings. In addition, some patients are also able to receive stem cell implants coming from cord blood. It can be difficult to find a matched donor for transplant treatment, and the treatments can pose significant risks. There’s no need to make something that’s already difficult seem more difficult.
The story would be stronger if it explicitly stated that there is no clear timetable for this treatment to become clinically available, if ever. That’s particularly true given language in the opening paragraph that calls this treatment “another win” for gene therapy. However, the story does provide enough information that readers can figure out the treatment isn’t available yet. Specifically, the story notes that “The main questions are how long the benefits will last, how much the treatment will cost if it wins approval, and whether it will replace stem cell transplants from donors….The U.S. Food and Drug Administration requires gene therapy participants to be monitored for 15 years, so these patients will continue to be studied, Williams said. Eight more patients are being added to the study as part of Bluebird’s plans to seek approval of the therapy in the U.S. and Europe” (emphasis added).
The story is sufficiently clear on this point to earn it a satisfactory rating. However, the New York Times story offers a deeper understanding of how this work developed over time and how it fits into the broader fields of gene therapy and ALD treatment.
The release incorporates information not found in the news release.
Systematic, Criteria-Driven
Monday at #ADA2021, an infectious disease epidemiologist, a journalist, and a researcher shared perspectives on communication of risk in the era of COVID-19: http://ow.ly/ROfR30rMeE7 @AstleyCM @garyschwitzer @berthahidalgo @ADA_DiabetesPro
Shoutouts to @susan_bewley @drkevinknopf @MichaelBaum11 in this call for more than fawning coverage from press releases for these kinds of stories.
Also, linked history inside goes back 5 years just on Grail apparently trying to become Holy Grail. https://twitter.com/garyschwitzer/status/1408849696416841731
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