Reporting on a very small (8 participants), very preliminary study evaluating the safety of stem cell therapy for cardiomyopathy (or heart failure), this story does a nice job describing the study and provides a good overview of the evaluation process a drug or treatment must undergo before it obtains FDA approval; however, the story could have been improved in a number of ways. For example, it would have been helpful had the writer provided more information on the safety outcomes of the study, included quotes from independent experts, and identified potential conflicts of interest. Furthermore, given the preliminary nature of this research and the fact that the purpose of the study was to evaluate safety rather than clinical efficacy, the story should not have included a quote from a study participant claiming that the stem cell treatment “almost certainly” saved his life.
Stem cells are a very promising area of research for a range of disease, but, so far, treatments for conditions such as heart failure remain elusive. A study this small should not be reason to give readers the false sense that therapies are right around the corner, and this story does a great job explaining the long road ahead. At the same time, very early studies like this should be presented with more critical analysis of the evidence and less effusiveness, especially a company that stands to benefit is involved in the study.
Since this treatment is not available, a discussion of costs is not warranted.
We appreciate that this is the rare story to point out that clinical significance was not considered, but we think the story leads readers down the wrong path by comparing a 25% reduction to a 5% reduction and not providing the absolute numbers. In addition, the story should have pointed out that the reduction was measured by comparing the patients’ baseline volume to their volume at the end of the study, as opposed to comparing their results to a similar group who did not receive the therapy.
As the story indicates, none of the participants experienced “significant side effects,” but it would have been helpful to define what “significant” means. For example, the study authors define serious side effects as death, non-fatal heart attack or stroke, hospitalization for worsening heart failure, blood flow compromise, or atrial fibrillation. While none of the subjects experienced these more serious side effects, the study did report that people had other issues such as premature ventricular complex, increased heart rhythm, and fluid buildup around the heart. Even when describing a study this small, the story should quantify the harms.
We always appreciate it when the lead of the story makes the small size of a study clear. This one described the study as “a small, preliminary human clinical trial.” It also does a nice job of describing the clinical trial phases that a drug or treatment must undergo before it can receive approval by the FDA. However, the story could have provided more information about the study participants, particularly that they were all men with a mean age of 57 years.
The story does not exaggerate the seriousness or prevalence of cardiomyopathy; however, it does include a quote from a study participant that makes an unsubstantiated claim regarding the treatment: “Almost certainly, I would be deceased or in much worse shape had I not had the opportunity to be in this program.”
This story does not interview any independent sources, nor does it reveal that some of the study authors received funding from BioCardia, the company that manufactures a device used to inject the stem cells into the participants’ hearts. In addition, the story should have noted that the study was funded in part by BioCardia.
The story compares the efficacy of stem cell therapy to medication and pacemakers, however, the comparisons are not adequately made and are done through the filter of the study authors, who may be biased given their financial ties to the industry.
This story stands out for taking readers through the application and approval process instead of just leading readers to believe that a therapy is around the corner. It says: “The next step is two more near-term studies. The first, which started in 2009, is a double-blind, placebo-controlled study of 60 patients designed further to test the safety of the procedure, but primarily its efficacy, or how well it works. Another study about to get underway will see whether bone marrow from a donor can work as well as the patient’s own bone marrow. Later, researchers will conduct a study involving 50 to 100 hospitals and many hundreds of patients aimed at winning final FDA approval.”
The story quotes the study author as saying that researchers have been investigating stem cell therapy for the treatment of cardiomyopathy for “close to a decade,” but to establish the novelty, the story should have explained why this tiny trial was a significant milestone in this field of research.
This piece does not rely on a press release.