This story is one of two that we are reviewing (the other is from the Associated Press) 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 New York Times story does a good job of describing everything that led up to the study, does a fair job of describing the study itself (and its results), and highlights the potential price tag of this treatment. However, the story doesn’t make clear how far this treatment is removed from widespread clinical use or the outstanding questions that remain about the treatment — such as the fact that we don’t know if the benefits are temporary.
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 tackles this issue head on, though what they report may not be comforting for those affected. Here’s how they address the issue: “The results of the new study also give rise to a concern that is becoming a regular feature of gene therapy work and other new biotech therapies: How much will this treatment cost? Bluebird Bio is not saying…[but one scientist involved in the study] expects the price to be similar to the hundreds of thousands of dollars it costs for a bone-marrow transplant.”
The story describes the outcomes for all 17 patients who were enrolled in the study, 15 of whom did not have symptoms of ALD after two years.
Potential adverse effects were not adequately addressed. The story did do a good job of highlighting concerns about gene therapies in general, pointing to problems that have cropped up in previous, unrelated gene therapy trials. And it mentioned that bone marrow transplants have risks. However, it didn’t address potential harms associated with this study in particular. 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. It’s not clear what effect, if any, the treatment had on the relevant patient.
The story does an adequate job of describing the study, but it’s missing at least one key point: Namely, the story doesn’t make clear that the study can’t prove ALD is “cured” in these patients — a longer follow-up time is needed to determine what the long-term impact of the gene therapy treatment will be. We also don’t know whether there will be significant adverse effects from the therapy (even if it is curative) in the long run.
The story includes input from multiple independent sources, which is good. The story also notes that the relevant study was funded (in part) by Bluebird Bio — a company that hopes to market the relevant gene therapy.
However, the story does not note that the principal investigator for the study, David Williams, has a conflict 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.
The story did a good job of describing other treatment options — including bone marrow and cord blood transplants — as well as the associated risks.
The story doesn’t make clear that this treatment is, at the very least, years away from clinical use. The story notes only that Bluebird Bio is “in hopes of marketing gene therapy for ALD.” This line, found more than halfway through a 1,300 word story, isn’t sufficient. This is an area where the Associated Press story fared better, noting that the FDA “requires gene therapy participants to be monitored for 15 years, so these patients will continue to be studied.” The AP story also notes that Bluebird Bio “plans to seek approval of the therapy in the U.S. and Europe.” The AP was stronger on this criterion.
This is a strong point for the story, which provides a fairly detailed background on how this treatment technique developed over time, how the treatment works and what sets it apart from other treatment options.
The story goes well beyond a news release.
Systematic, Criteria-Driven
Puleeeeease... this 'game changer' weight loss drug causes more nausea and vomiting than a placebo. Some game, some change. My friend Gary would spank you for using the eighth word medical reporters should never, ever use. @garyschwitzer @thackerpd
https://www.healthnewsreview.org/toolkit/just-journalists-writing-tips-case-studies/7-words-and-more-you-shouldnt-use-in-medical-news/ https://twitter.com/OttawaCitizen/status/1361426796126830597
A scary @statnews Op-Ed warns that govt attempts to rein in Pharma may cut off access to "life-changing" drugs.
It takes @garyschwitzer to let us know: the author spent 10 years as Pfizer's public-policy chief. https://twitter.com/garyschwitzer/status/1360325022578053123
.@TranspariMED criticizes STAT op-ed for not disclosing pharma funding of author’s institute - similar to criticism we’ve made of STAT op-eds in the past. @transparify @ThinkTankWatch @icer_review https://www.healthnewsreview.org/2021/02/transparency-watchdog-criticizes-stats-non-disclosure-on-pro-pharma-op-ed/
Comments
Please note, comments are no longer published through this website. All previously made comments are still archived and available for viewing through select posts.
Our Comments Policy
But before leaving a comment, please review these notes about our policy.
You are responsible for any comments you leave on this site.
This site is primarily a forum for discussion about the quality (or lack thereof) in journalism or other media messages (advertising, marketing, public relations, medical journals, etc.) It is not intended to be a forum for definitive discussions about medicine or science.
We will delete comments that include personal attacks, unfounded allegations, unverified claims, product pitches, profanity or any from anyone who does not list a full name and a functioning email address. We will also end any thread of repetitive comments. We don”t give medical advice so we won”t respond to questions asking for it.
We don”t have sufficient staffing to contact each commenter who left such a message. If you have a question about why your comment was edited or removed, you can email us at feedback@healthnewsreview.org.
There has been a recent burst of attention to troubles with many comments left on science and science news/communication websites. Read “Online science comments: trolls, trash and treasure.”
The authors of the Retraction Watch comments policy urge commenters:
We”re also concerned about anonymous comments. We ask that all commenters leave their full name and provide an actual email address in case we feel we need to contact them. We may delete any comment left by someone who does not leave their name and a legitimate email address.
And, as noted, product pitches of any sort – pushing treatments, tests, products, procedures, physicians, medical centers, books, websites – are likely to be deleted. We don”t accept advertising on this site and are not going to give it away free.
The ability to leave comments expires after a certain period of time. So you may find that you’re unable to leave a comment on an article that is more than a few months old.
You might also like