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
The tale of two “negative” studies. Or, how spin is applied to make research findings look more impressive. A must read. https://johnmandrola.substack.com/p/the-tale-of-two-negative-studies
The @nytimes chose to interview one of the editors of the @AnnalsofIM (publisher of this paper) who "gushed with extraordinary glee: It’s huge! There are very few things that reduce your mortality by 30%."
Turns out coffee is not one of those few things despite all that glee. https://twitter.com/garyschwitzer/status/1533202075928215558
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