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Gene Therapy Shows Potential Against Heart Failure


2 Star


Gene Therapy Shows Potential Against Heart Failure

Our Review Summary

Are we really going to report commonly now when early research moves from one phase into another? What researchers announced was that a year after a very small number of heart failure patients were given a type of gene therapy intended to improve how their heart muscles manage calcium cycling there appear to be enough indications of relative benefits and safety to move ahead to a phase 3 trial that would be capable of providing more reliable evidence of clinical effects. What’s more, the key conclusions of this trial were announced at another heart failure meeting last summer. This presentation just updated the statistics with a few more months of data.

Instead of asking probing questions of researchers, the story relies on quotes from a news release. Instead of getting comments from independent sources that illuminate the specific strengths and weaknesses of this trial, the story just passes along general statements about heart failure and the preliminary nature of research.

At least the story discloses the use of prepared statements and it notes that the company developing this gene therapy funded the trial.


Why This Matters

Earlier reports about this trial highlighted questions about the statistical methods used by the researchers in this trial… and words of caution about reading too much into the findings. This story could have done a better job explaining to readers that this trial was capable only of determining whether or not this gene therapy is ready for the next phase of testing – and that it cannot settle questions about whether the therapy is safe and effective.


Does the story adequately discuss the costs of the intervention?

Not Applicable

Although this gene therapy has barely begun the long road of clinical trials and so we won’t hold this story to the expectation that it discuss costs, the story could have offered readers at least some general comments about how the costs of this sort of gene therapy might compare with conventional treatments for heart failure.

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

Not Satisfactory

This story doesn’t seem to distinguish between “getting better” and “not declining as fast.” As one line states, “At both six months and a year later, the patients who had received the new gene saw their risk for death, cardiac transplantation, worsening heart failure and hospitalization decline by half.” That sentence seems to say that the patients had lower risks than before they enrolled in trial, when what researchers actually reported was that the patients given the gene therapy were only half as likely as the patients who got a placebo to have one of those bad events.

The story reports only relative benefits without telling readers the absolute numbers. One result reported in a company news release was particularly striking, that during the year following treatment, the average number of days spent in the hospital was 0.4 per patient for the 9 patients who got the highest dose and 4.5 days per patient for the 14 who received a placebo. The story could have at least included the actual numbers of deaths reported in each group.

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

Not Satisfactory

The story does not mention any harms. Even though the researchers said in their meeting abstract that the therapy had “an excellent safety profile” in this trial, the story should have pointed out that patients have been harmed and some have died during experiments using other types of gene therapy.

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

Not Satisfactory

The story falls short in accurately describing both the big picture and key details. For instance, the lead sentence says the gene therapy partially restored “the heart’s ability to pump in 39 heart failure patients.” But 39 patients was the total number enrolled in the trial. Of those, 14 received a placebo. Of the 25 who received the gene therapy, only 9 were given the high dose that produced results the researchers highlighted.

As for the big picture, not only was the purpose of this trial limited to determining if the therapy appears to be safe and effective enough in order to be given to a much larger number of patients in a trial capable of producing convincing evidence of clinical effects, but the statistical methods used have sparked debate among observers. Following the release of shorter-term results last summer, one independent observer was quoted as having reservations about the way the trial defined improvement, which he said had never been used before and hadn’t been well validated. The generic statement in the story that results are preliminary and need more investigation isn’t enough to put the sketchy nature of these results into the proper context for readers. At least the story included a standard disclaimer that “research presented at meetings isn’t subjected to the same level of scrutiny as studies published in peer-reviewed journals.”

See “CUPID: First-in-human gene therapy for advanced heart failure promising in small study”
available at

Does the story commit disease-mongering?

Not Satisfactory

Although a quote from one of the researchers and another line in the body of the story refer to patients with “advanced heart failure,” the headline and lead of the story refer to just “heart failure,” which includes a far broader range of people. A company news release defined the trial participants has having moderate to severe heart failure and significantly impaired pumping function of their hearts and less than half the normal ability to transport and utilize oxygen during exercise testing. The story should have been clear from the top about the severity of disease in the participants in this trial. A single additional sentence would have gone a long way in addressing this.

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


The story does quote two independent experts and reports that the trial was funded by the company developing this gene therapy. Unfortunately, those experts’ quotes didn’t add much to help readers evaluate the quality and value of this trial. But it nonetheless gets a barely passing grade on this criterion.

Does the story compare the new approach with existing alternatives?

Not Satisfactory

Other than saying that one goal of this line of research is to provide an alternative to heart transplants, the story does not describe the alternative treatments available to people with heart failure. For example, even when drug therapy loses its effectiveness, some heart failure patients are receiving heart assist pumps for long-term use… so there are approved alternatives to a heart transplant for some patients like those in this trial. The story should have included that fact.

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


The story clearly states that this gene therapy is experimental and not available to patients.

Does the story establish the true novelty of the approach?


This gene therapy technique is indeed something new, but the story would have been better if it had included some independent assessment of a researcher’s claim that, “This is the first time gene therapy has been tested and shown to improve outcomes for patients with advanced heart failure.”

Also, the researchers reported very similar results at other heart failure meetings last summer. The story does not make clear that this latest announcement merely adds a few more months of follow-up, not entirely new results.

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

Not Satisfactory

At least the story discloses that the quotes from the lead research are taken from a university news release.

Total Score: 3 of 9 Satisfactory


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