CBS cures two diseases in one 400-word story

Does the story adequately discuss the costs of the intervention?

Not Satisfactory

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.

Does the story adequately quantify the benefits of the treatment/test/product/procedure?

Not Satisfactory

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.

Does the story adequately explain/quantify the harms of the intervention?

Not Satisfactory

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.

Does the story seem to grasp the quality of the evidence?

Not Satisfactory

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.

Does the story use independent sources and identify conflicts of interest?

Not Satisfactory

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.

Does the story compare the new approach with existing alternatives?

Not Satisfactory

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.

Does the story establish the availability of the treatment/test/product/procedure?

Not Satisfactory

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.

Does the story establish the true novelty of the approach?

Not Satisfactory

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.