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Stem cell therapy for macular degeneration


3 Star


Stem cell therapy for macular degeneration

Our Review Summary

It’s a tough thing for a reporter to write about a study that hasn’t been conducted yet. Without any actual data to work with, the temptation is talk too much about what the researchers hope they will find in the study; discussion of harms and other caveats often gets overwhelmed by this optimistic speculation. The story we review here didn’t succumb to the worst excesses that these kinds of reports are subject to, and it did some things very well. For example, when discussing the new treatment that’s set to be studied — an embryonic stem cell therapy for age-related macular degeneration (AMD) — it gave some interesting and detailed background on how AMD develops and how the new treatment might work. But in the end, we thought the discussion of hypothetical benefits was out of balance considering the very early stage of the research, and there was no mention of potential costs or harms. The story also didn’t explain carefully enough who might be a candidate for this treatment, should it eventually make it to market.


Why This Matters

There are no effective treatment options for the dry form of AMD, which is a leading cause of vision loss in older adults. The use of embryonic stem cells represents an exciting new approach to this disease, but given the early stage of development for such therapies, news coverage needs to pay appropriate respect to cautions and caveats about the research. Otherwise such stories may provide readers with false hope instead of information.


Does the story adequately discuss the costs of the intervention?

Not Satisfactory

Although it’s too early in the game to know exactly what this treatment is going to cost, our rule of thumb is to expect some kind of cost discussion whenever there is talk of benefits. This story didn’t meet that standard. The story could at least have said that we do not know how much the new treatment will cost, but that it is likely to be expensive.

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

Not Satisfactory

With the lack of any real data to discuss, this story needed to be exceptionally disciplined to avoid pumping up hypothetical benefits. But the speculation in this story, while relatively restrained, is still out of balance considering the early stage of the research.  In one passage, the story relays company hopes that the new treatment might “keep vision intact — or even restore it to some degree.” Later, it suggests that the treatment might “buy time for patients, delaying vision loss for perhaps years.” Such speculation might have been acceptable had the story provided a more forceful and sober assessment of the slim chances this therapy has of ever reaching the market. As it stands, though, the coverage unduly raises hopes for what remains a longshot bid at successful treatment.

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

Not Satisfactory

Injecting substances into the eye is bound to cause problems for some people, but this story doesn’t mention any possible harms. This is especially troublesome considering that safety assessment is the primary objective of a phase I study. Other treatments involving eye injections are associated with increased risk of potentially serious eye infections, which would seem to be a potential risk here. In addition, animal studies suggest that undifferentiated embryonic stem cells can cause a certain type of tumor known as a teratoma. While the treatment being offered here is supposed to include only differentiated stem cells, which presumably don’t carry this risk, the point again is that we just don’t know whether or not the treatment is safe — hence the need for the study.

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

Not Satisfactory

Since the study discussed in the story has yet to be conducted, there’s really not much evidence to evaluate. However, the story makes the most of what it has to work with. It does an admirable job of explaining the physiology of the macula and how AMD develops. It also presents some animal data suggesting that there is reason to believe the approach being studied has merit. The story cautions that success in these animal studies “doesn’t always translate to humans,” and it notes that it will be especially tough for new cells to take hold in the eyes of older patients. The story also does a reasonable job of describing the design of the new study and emphasizing its focus on safety.

What the story fails to adequately explain is what kinds of patients might be candidates for the new treatment. Although it takes considerable pains to explain the differences between the “wet” and “dry” forms of AMD, it never explains that the new treatment is targeted at the more common dry form of the disease, which usually is slower to progress and less severe than wet AMD. The upshot is that the story may generate false hope for individuals with wet AMD, which often causes significant vision loss.

We also thought it was potentially confusing to readers that the story led with an estimate of about 10 million Americans who suffer some degree of vision loss from age-related macular degeneration, then jumping to the fact that the company developed this approach to treat a rare childhood version, but then jumping to the assertion (even though the trials haven’t been done) that if this works, the cells would have a much bigger effect for the age-related problems.  We think it’s best to be cautious about predictions of efficacy for a problem that hasn’t been treated yet – especially when the preliminary proof-of-concept human trials haven’t even been done.

A close call here given the excellent job in some areas, but the failure to describe appropriate treatment candidates is a significant gap that warrants an unsatisfactory.

Does the story commit disease-mongering?


Although this story should have been more clear about who might be a candidate for this treatment (an issue discussed more thoroughly under the Evidence criterion below), the tone of the story was reasonably cautious and we don’t think it crossed the line to disease-mongering.

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


Two expert sources not affiliated with the research are quoted. As far as we are aware, there are no conflicts that weren’t disclosed to the reader.

Does the story compare the new approach with existing alternatives?

Not Satisfactory

While treatment options are limited, individuals with dry AMD may benefit from taking a supplement containing a specific formulation of vitamins. This has been shown to help prevent intermediate dry AMD from progressing to severe AMD. There are also several effective therapies, including drugs and laser treatments, for the wet form of AMD. It wouldn’t have required much time or space for the story to at least mention these.

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


The story makes it clear that this treatment is in the early stages of experimental research. It doesn’t speculate inappropriately about when the average patient might have access to it.

Does the story establish the true novelty of the approach?


The use of stem cells to treat macular degeneration is just being translated from mice to humans, so the story is correct to characterize the treatment as novel. It could have noted that the treatment discussed in this story is just one of many new approaches researchers are studying.

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


The story features interviews with two independent experts, so we can be sure it didn’t rely on a news release.

Total Score: 5 of 10 Satisfactory


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