The approach to Duchenne muscular dystrophy described in the story is new and offers a potential approach to a host of genetic disorders.
Because the science is new and has not been studied in great detail, there are no guarantees that the approach will work in the long run and that side effects will not limit its usefulness. This story did a reasonable job in describing a study based on information provided by the manufacturer and not yet published or even in abstract form.
Reporting of clinical trial results should always be balanced and in context. This is even more important when reporting about possible treatments for devastating diseases like Duchenne muscular dystrophy.
Not applicable. The drug is in early stages of development and as such, identifying the potential cost is premature
The story provided just enough caveats to the positive aspect of the study results. “The study measured how far the boys could walk in six minutes. Those who received the higher dose walked an average of 21 meters farther after 48 weeks of treatment than at the beginning of the trial, a gain of about 5 percent. The boys in the placebo group walked 68 fewer meters at the end than at the beginning, a decline of about 17 percent. One possible reason for skepticism is that two of the boys on a lower dose of the drug rapidly lost the ability to walk, even though the level of dystrophin in their muscles increased substantially.”
The story added a key piece of information at the end, “Eteplirsen actually turns off just one part of the dystrophin gene, known as exon 51, which contains a mutation that in some patients stops production of dystrophin. By skipping over this exon, muscle cells can produce an imperfect form of dystrophin that nonetheless is at least partly functional.
The story provided some limited information on what is known about the potential harms, “It is also unclear how long the effects of the drug would last or if safety issues would arise with longer treatment.” “Eteplirsen, which Sarepta says has shown no side effects so far,…”
The company’s press release noted: “The safety profile of eteplirsen was evaluated across all subjects through 48 weeks and there were no treatment-related adverse events, no serious adverse events, and no discontinuations. Furthermore, no clinically significant treatment-related changes were detected on any safety laboratory parameters, including several biomarkers for renal function. ” We think that this information should have been included in the story, as it would have provided more meaningful details to back up the sparse comments on harms that were published. No, we don’t expect a story to read like a journal. But we do draw a line between what we think is meaningful explanation to readers and what isn’t.
The story provided the reader with the caveats necessary to put the information into context. “There are many caveats. The trial had only 12 patients, with only four receiving the high dose and four the placebo, and the data has not been reviewed by experts. It is also unclear how long the effects of the drug would last…” One possible reason for skepticism is that two of the boys on a lower dose of the drug rapidly lost the ability to walk, even though the level of dystrophin in their muscles increased substantially.
The story is based on a pre-abstract release of information from the company. The study’s principal investigator, the company CEO and parents of children enrolled in the study are the only sources quoted in the story. We think that an independent expert should have been quoted.
The story noted that there is at least one other drug in development and that there is a rather ineffective treatment available in the form of steroids.
The story makes it clear that the drug is investigational and not available at the present. It also provided some additional insight, “But Ms. McNary said Sarepta had refused to provide the drug to Max’s 13-year-old brother, Austin, who could not be in the trial because he already could not walk.” “Mr. Garabedian said Sarepta did not have the money or the manufacturing capacity to make the drug available outside of clinical trials. “That would be the quickest path to insolvency,” he said.“
The story makes it clear that this is new and novel in the treatment of Duchenne muscular dystrophy
The story does not appear to rely on a press release.
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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