This 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.
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.
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.
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.
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.
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.
There’s no disease mongering here. It’s hard to overstate the devastation of Duchenne muscular dystrophy.
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.
Given the scarcity of effective treatments for this disease, a comparison with other treatment options is probably not warranted.
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.
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.
It does not appear that this story is based on a news release.
Systematic, Criteria-Driven
Come on, NY Times; you must do better than this. Important research doesn’t need hype and the “breaking news” BS doesn’t help readers or patients. https://www.healthnewsreview.org/2022/03/nyt-proclaims-breaking-news/
Our editorial team was blessed to have Sharon as a contributor during our best years. As this obit states, she was “kind, patient, wise, thoughtful and generous.” Thank you, Sharon; now rest in peace. https://journalism.wisc.edu/news/in-memoriam-sharon-dunwoody-1947-2022/
We’ve written about the hype of liquid biopsies for years (see link in comment below). And now this…. https://www.statnews.com/2022/01/12/medicare-shouldnt-cover-liquid-biopsies-early-cancer-detection/
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