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ABC joins bandwagon, puts rosy spin on stem cell therapy, this time for muscular dystrophy

Teen Feels 'Hopeful' After Experimental Stem Cell Procedure on Damaged Heart

Our Review Summary

iStock_000055399860_SmallThis is a story about one young man receiving an experimental infusion of stem cells into his heart, to help with tissue scarring due to muscular dystrophy. Despite booming research around potential stem cell treatments for a wide range of conditions, the list of diseases for which stem cell therapy has been shown to be effective remains very short, according to the International Society for Stem Cell Research. Yet media outlets often put an optimistic spin on unproven stem cell therapies, as we’ve seen in our examinations of stories on heart failure, chronic obstructive pulmonary disease, severe combined immunodeficiency, and multiple sclerosis.

Showcasing a few preliminary cases with encouraging outcomes can exaggerate the potential benefits of a treatment and raise false hopes for patients. This story, based on a single case with an unknown outcome, fits this pattern. It fails to counter rosy statements by a physician and a patient with realistic assessments of the potential benefits and pitfalls of an unproven procedure for a fatal disease with no known cure. The story also lacks clinical details that would help readers get a true picture of the nature of the treatment, as well as a discussion of costs.

 

Why This Matters

Duchenne muscular dystrophy (DMD) is a heartbreaking disease that affects an estimated 1 in 3,600 boys as well as some girls. The most common inherited pediatric muscle disorder, it’s caused by a shortage of a protein called dystrophin, which leads to progressive muscle weakness. Most patients lose their ability to walk by age 12, and average life expectancy is about 25. Often patients die of heart failure because dystrophin deficiency leads to cardiomyopathy, a weakness of the heart muscle that makes the heart less able to pump blood and maintain a regular rhythm. A recent review of current therapies states that “only steroids have been shown to produce a slowing in the declining course of the disease.” Recent research has focused on using stem cells and gene therapy to create new, healthy muscle cells and thus halt or reverse the disease.  The rapid advance and certain fatality of Duchenne muscular dystrophy has patients and their families desperate for new treatment options.  Reporters have an obligation to those families to present stories in a reasonable and responsible fashion.

Criteria

Does the story adequately discuss the costs of the intervention?

Not Satisfactory

The cost of this procedure and the potential for health insurance coverage are not addressed. The costs of high-tech interventions such as stem cell infusions are likely to be high and given their experimental nature are not likely to be covered by insurance.

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

Not Satisfactory

Quantifying long term benefits at this point may not be possible. The story offers this statement from a physician: “If we can preserve his cardiac function he has a fantastic outlook.” But the story missed an opportunity to educate readers by explaining how physicians intend to measure results. The story fails to quantify any improvement in cardiac function in this young man. Previous clinical trials of stem cell infusions in patients with hearts damaged by heart attack have shown modest improvement in function using standard diagnostic methods in the short term.

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

Not Satisfactory

Most patients with Duchenne muscular dystrophy would probably be willing to take almost any risk for the chance of being cured. Still, any stem cell therapy can be perilous. According to the International Society of Stem Cell Research, potential risks include complications that create short-term and long-term health problems, ineligibility for future clinical trials or treatment options, and time away from friends and family. The story could have offered readers insight by explaining what was involved from the patient’s standpoint: how long the procedure took, what discomfort it may have presented, what follow-up is involved, etc.

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

Not Satisfactory

The story asserts that the patient, Caleb Sizemore, is the first to receive an infusion of stem cells in his heart in the hope of stopping or reversing scarring related to Duchenne muscular dystrophy. It does not discuss any similar procedures that may have been used to treat other conditions and how they have fared. Optimistic quotes from a physician and a patient are not balanced with a detailed explanation of the significant hurdles that must be overcome before stem cell therapy could be counted as a successful treatment for Duchenne muscular dystrophy. The story only points out that Sizemore will return to Cincinnati from his home state of North Carolina “in a few months” so physicians can determine whether the procedure had an effect. The story skimps on significant clinical details, such as what type of stem cells were used.

Does the story commit disease-mongering?

Satisfactory

There’s no disease mongering here. It’s hard to overstate the devastation of Duchenne muscular dystrophy.

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

Not Satisfactory

This story begs for comments from outside experts, including research clinicians and patient advocates who could provide unbiased perspective on the potential for this treatment and describe how it fits into ongoing research in the field. For example, outside experts might shed light on the outcomes of similar procedures that have been performed on cardiac patients and whether they are relevant to this case.

Does the story compare the new approach with existing alternatives?

Not Applicable

Given the scarcity of effective treatments for this disease, a comparison with other treatment options is probably not warranted.

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

Not Satisfactory

The story leaves the misleading impression that widespread application for millions of patients could be around the corner. It paraphrases a physician as stating, rather confusingly, that “if the medication works on patients like Sizemore, it could then be tested on millions of other Americans who have scarring on their heart due to disease or past heart attacks.” Moreover, Sizemore it quoted as saying, “It is very exciting especially because of the impact it has on other kids and how it paves the way so that others can get the treatment and how this will change people’s lives and extend them.” These statements are a huge leap, begging for a discussion of the complexity of stem cell therapies and the process of measuring patient outcomes and proving efficacy.

Does the story establish the true novelty of the approach?

Satisfactory

There is no reason to believe the story is inaccurate when it says identifies its subject as the first patient to have had a stem cell infusion for Duchenne muscular dystrophy, though it should have cited a source. As previously mentioned, it appears that similar procedures have been used on cardiac patients, although the story lacks sufficient details to tell for sure.

Does the story appear to rely solely or largely on a news release?

Satisfactory

It does not appear that this story is based on a news release.

Total Score: 3 of 9 Satisfactory

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