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Gene therapy successful on a blindness


5 Star

Gene therapy successful on a blindness

Our Review Summary

The main news value of this trial is that it reports some improvement in some patients – in contrast to so many gene therapy trials that have failed to produce beneficial results.

This story includes a number of clear warnings about the preliminary nature of the experimental attempt to correct a genetic mutation that leads to blindness. While the story delivered the emotional power of the story of a boy who can play outside now that his vision has improved, readers are not introduced to any of the other trial participants, who apparently experienced more modest improvements, if any.

The story did not provide sufficient information on the potential toxicities of gene therapy or in the risks associated with the particular treatment.

It also should have made clear how rare this mutation is and that even if this specific gene therapy treatment is eventually approved, it may be appropriate for only a few hundred people nationwide. The larger significance of the trial is that it may encourage further exploration of treatments based on similar principles. 

Overall the story provides great human interest without compromising the integrity of the research and its limitations at the present time. 


Does the story adequately discuss the costs of the intervention?

Not Applicable

This experimental treatment is still very far from clinical use. However, the commentary that accompanied the research article in the Lancet referred to the difficulty and high cost of identifying children with this gene mutation. A mention of that issue would have helped readers to understand better how much work remains before it is known whether this approach could eventually become a practical treatment.

The story does point out that this trial cost more than $2 million and provided data on just a dozen individuals.

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


Highlighting the apparent benefit to one young man along with clear information on the other 11 participants in the trial appears reasonable and provides the reader with a goodunderstanding of the level of benefit overall.   While the emphasis is on a single patient, the story is reasonably well balanced. 

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

Not Satisfactory

The story does not report the temporary retinal detachments or other complications experienced by some of the trial participants. It does not address the unknowns or potential risks of the experimental treatment other than to note that the virus used to deliver the gene therapy in this trial is different from the one that was linked to the death of a patient in an unrelated gene therapy trial a decade ago.

Although the researchers reported the patients in this small trial did not suffer any “serious adverse events,” the story does not include any discussion of what potential harms might be encountered as the procedure is performed on more people.

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


Overall, the story does make clear that the research is at an early stage and that far more work remains. However, the caveats are overshadowed by the headline proclaiming the therapy “successful” and the lead paragraphs focusing on the improvement seen in just one child who apparently improved more than most of the trial participants.

Does the story commit disease-mongering?


The story does not exaggerate the effects of the progressive blindness known as Leber’s congenital amaurosis. However, readers are likely to overestimate the potential of this specific gene therapy because the story fails to point out that Leber’s congenital amaurosis affects only 2 or 3 of every 100,000 people in the U.S. and (because the specific gene mutation relevant to this trial may account for only 6 percent of the cases) there may be only a few hundred people nationwide who have the same gene mutation as those enrolled in this study.

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


The story includes comments from a researcher who was not involved in this trial. It points out that the major funder was the hospital-based research center where several of the researchers work. It is not clear from this story who owns the rights to development and sales of the treatment if it eventually is approved. The journal article notes that two of the researchers hold pending patent rights, but have released their financial rights.

Does the story compare the new approach with existing alternatives?

Not Applicable

There are no alternative treatments for Leber’s congenital amaurosis. Patients may use assistive devices that enhance their limited vision.

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


The story points out that researchers are years away from even seeking approval for clinical use of this gene therapy technique.

Does the story establish the true novelty of the approach?


The novelty of positive outcomes, any positive outcomes, in a gene therapy trial, rather than the prospect of near-term or widespread clinical application, is indeed the news here.

The story notes that this report is a follow up to an earlier publication and that other teams of researchers have also reported improvements in similar patients.

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


The story does not appear to rely on a news release. It notes that the featured patient and his parents were introduced to reporters at a hospital news conference.

Total Score: 7 of 8 Satisfactory


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