Stem cell therapy for macular degeneration

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 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.