This 400-word story is an update of a 2014 news release from UCLA that provides little in the way of new information about a new stem cell treatment for severe combined immunodeficiency (SCID). Given the story’s brevity, many key aspects of the disorder and its current treatment were neglected. The research methods were not clearly articulated and the fact that the trials thus far have been phase 1-2 clinical trials all take away from the importance of the research itself. The last statement concerning the use of the approach as a “cure” for sickle cell disease is an unsubstantiated add-on.
SCID is a rare but important genetic disorder that has been managed at children’s specialty hospitals for many years. It would have been great had this story provided some data regarding the rarity of the condition. Bone marrow transplant or simple replacement of adenosine deaminase (ADA) has been the standard approach. The new approach described in the story may prove to be an important advancement in the treatment of these children but a more measured tone would have been appreciated. The results to date are certainly encouraging but the method has a long way to go to routine clinical application. To suggest, as the headline does, that this treatment “looks like a cure” is a bit of an overstatement at the moment.
We think that costs should almost always be part of a discussion for a new treatment and this brief report is no exception. Autologous stem cell transplants, performed for certain types of cancer, can cost in excess of $100,000. Since the procedure is experimental, as noted in the story, it is not likely to be covered by insurance.
The story relies heavily on one child’s journey and notes that ” …the treatment has restored immune systems in all 23 patients in the most recent clinical trials.” But we aren’t told how long these children were followed after the procedure or what a “restored immune system” is or how it was measured. There is simply too little information provided to assess this treatment’s potential.
Harms were not discussed in any way. With such a serious disease, harms would have to be significant to outweigh the potential benefits, but they should still be mentioned.
The story’s depiction of the procedure doesn’t convey the full scope of what’s involved. It states, “….the treatment involves taking bone marrow from the patient to gather stem cells. A cloned gene is then added to correct what was missing at birth…”Those stem cells are given back to the patient where they can go back to the bone marrow and make the blood cells for the rest of the patient’s life.” Although it sounds pretty straightforward, a look at the actual procedure as listed at ClinicalTrials.Gov provides a more complete picture. The stem cells are collected under general anesthesia and treatment with busulfan (which can cause significant side effects) to reduce bone marrow cell production in preparation for the stem cell infusion.
There is no description or assessment of the study design. ClinicalTrials.gov lists the existing research protocol as a phase 1-2 trial. So the primary purpose of the study is safety and not effectiveness. We are not provided with any of the specifics of the methods, inclusion and exclusion criteria, or much about the follow-up period.
No disease mongering is evident here. However, this disease is serious and often fatal. There could have been more information provided such as the average life expectancy without treatment and with alternative treatments, to put the disease in context and not just say it is serious.
The only two people quoted are the clinical investigator and the parent of a child treated. A comment or two from other experts in the field would have rounded out the reporting.
Most children with SCID receive a bone marrow transplant or receive weekly injections of ADA. This fact was not noted in the story. In addition a quick check of ClinicalTrials.Gov uncovered several new approaches that are technically different from that of the story. In addition, drug companies are working on the development of other approaches to this form of SCID.
The story notes, “Dr. Kohn is currently working with the FDA to make his treatment available nationwide. He’s also testing the same method as a cure for sickle cell disease. Clinical trials for that treatment are now underway.” This gives the impression that although the approach is still experimental, its availability nationally is a forgone conclusion. In fact, many treatments get to this stage and do not pan out.
As we noted previously, most children with SCID undergo a bone marrow transplant. The use of gene modification for the disorder is more than 20 years old having started in France in the early 1990s. The approached highlighted in the story is indeed new but the general approach has been around for some time.
We couldn’t find any recent news release that this story might have been based on. And although it’s similar to a 2014 news release that we identified above, it has quotes that are different and appear to reflect original reporting. We’ll give the benefit of the doubt here.
Systematic, Criteria-Driven
We are all worse off for not having @garyschwitzer's excellent, daily health care reporting, but his story today should sound the alarms and be a warning to anyone who gets health care information from local TV health news (read: much of America) (1/2) https://www.healthnewsreview.org/2019/11/why-hire-a-full-time-health-reporter-when-you-can-buy-news-off-the-shelf/#.Xeank0LWjyA.twitter
You can’t know what navigating #Medicare will be like until you get into it. Here’s advice for journalists who report on health care. Help from @Trudy_Lieberman. We need more like her. https://www.healthnewsreview.org/2019/12/after-a-year-on-medicare-advice-for-journalists-on-stories-that-need-to-be-told/
Even if you don't **intentionally** watch health segments on TV news (you know, like when it's on in an airport or gym or . . . ) you should read this warning from @garyschwitzer : https://www.healthnewsreview.org/2019/11/why-hire-a-full-time-health-reporter-when-you-can-buy-news-off-the-shelf/ #badjournalism
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