This HealthDay story reports on the very early results of a phase one trial of a drug, cimaglermin, intended to improve the left ventricular function among patients with a type of heart failure known as “severe left ventricular systolic dysfunction.”
The story does clearly say that it is a phase one trial and explains that clinical use of the drug may be years away, if then. The story would have been stronger if it had detailed exactly what the researchers were measuring when they concluded the drug worked better than placebo.
Obviously, if a new drug is found that works effectively to thwart this kind of heart disease, then that’s good news all around, especially if the drug is needed less frequently and is well tolerated, as the story suggests. But care needs to be taken in potentially over-touting the results of these very early trials, and therefore raising the hopes of patients who may be disappointed at a later stage in the research process.
Only larger studies with longer follow-up will determine whether these very preliminary results are supported or not. A key outcome to follow will be preventing admissions to the hospital for heart failure, and ultimately, prolonging life.
There is no mention of the cost for the tested drug, cimaglermin, in this story. The costs for regularly prescribed drugs for the medical condition that’s the focus of the study — weakened left ventricular function — are well known and although cimaglermin is an experimental drug, it should be possible to predict whether the new drug would be comparatively priced, cheaper or more expensive. Readers would benefit from that knowledge.
While this story discusses that the drug seemed to benefit patients and was effective, it doesn’t say how that was measured, and by how much the measurement improved due to the drug.
(By digging into the research paper, via figure 3, we found that the benefits were measured by tracking absolute increases in ejection fraction, which is the amount of blood pumped out the ventricles. It increased 8% in the group receiving a medium or high dose of the medicine.)
One issue that might be confusing for readers: The story notes that a “phase 1 trial like this one is designed to see if a new drug is safe, not to test its effectiveness.” If that’s the case, then why is the framing of the story about how effective it was?
The story clearly points out that side effects included nausea and headaches, and that one participant did experience abnormal liver function after receiving the highest dose, a side effect that eventually cleared up.
This is a strong point of the story: It clearly establishes that this was a small phase one randomized controlled trial, and the story explains what that means for the drug’s development.
A phase 1 trial like this one is designed to see if a new drug is safe, not to test its effectiveness. Before cimaglermin could be used to treat patients, it must prove its worth in a series of progressively larger and challenging trials and then be approved by the U.S. Food and Drug Administration. The process can take several years. Based on these preliminary findings, larger trials are being planned, Lenihan said.
But, as noted in quantify benefits criterion above, readers might be confused by why so much of the story focused on the drug’s effectiveness, if this preliminary study wasn’t designed to test that.
The story doesn’t commit disease mongering.
The story does get credit for quoting a researcher not affiliated with this project, but it neglects to mention some obvious potential conflicts of interest among the members of the research team. According to the research paper, eight of the 13 researchers have some linkage to pharmaceutical firms and seven of them have a connection to the sponsor of the study, Acorda Therapeutics, Inc., either as consultants, employees, or stockholders, and two of them have had research funded by the company.
The story does mention alternatives for dealing with weakened left ventricular function, primarily through this paragraph:
“People with heart failure often take a combination of drugs, Lenihan said. These include medications to lower blood pressure and diuretics to help remove excess fluid that builds up as a result of the heart’s labored pumping ability. In addition, some people have implanted defibrillators or pacemakers.”
We’ll rate this category satisfactory, given that the story admits that it is reporting on a phase one trial, and includes the following information:
“Before cimaglermin could be used to treat patients, it must prove its worth in a series of progressively larger and challenging trials and then be approved by the U.S. Food and Drug Administration. The process can take several years.”
The drug is clearly described in terms of its experimental nature at this point.
This story doesn’t appear to have relied on a news release.