This story discusses research findings about a drug that may slow the progression of geographic atrophy, a type of macular degeneration that leads to vision loss.
The story provides a careful, reader-friendly explanation of the mechanism by which lampalizumab is likely to work, and a concluding section with clear information about what can currently be done for geographic atrophy. However, the article falls short in precisely describing the method and findings of the study–and missed an important conflict of interest involving the drug company and a source.
Geographic atrophy, a type of advanced age-related macular degeneration (AMD), affects approximately one million Americans and more than 5 million people worldwide. It is characterized by gradual and irreversible vision loss. Geographic atrophy is not the most common type of AMD; neovascular macular degeneration is more prevalent. However, a number of treatment options are available for the latter, whereas no treatment is currently available for geographic atrophy. Therefore, the condition represents a critical, unmet medical need.
This study indicates a possible new treatment, though the measured benefit was pretty small and it’s not clear if it helped anyone see anyone better or was worth the risk of side effects. Readers should also keep in mind that this drug is an immunotherapy–all of which are very expensive (at least until generics come along), so it’s also likely to be very expensive if it’s approved.
Even if we can’t know the exact cost of what this drug might be (if it’s approved–that’s TBD), we can surmise that it will be very high based on the price tags of every other drug in this class, known as immunotherapies. The story should have noted this.
The story tells us that patients who had the genetic variation and were treated with the drug had “44 percent less eye damage than the untreated patients.” However, since there is no information on what the baseline measurements were, we’re not sure what that means. Is this a little or a lot in absolute terms? Did the treated group have noticeably improved vision?
As the study shows, all patients got worse after 18 months. But the lesions in the gene-carrier placebo group grew by about 4.2 millimeters, whereas the treated gene-carrier group’s lesions grew 2.3 millimeters. It’s not clear at all if that provides any actual vision benefit–or is worth the risk of side effects, since presumably the patients will need to be treated indefinitely. We wish the story had explored this important aspect.
The story states that there were no “safety concerns” noticed during the trial. We’re not sure what that means–does that mean there were zero side effects? Or just no life-threatening ones? When we looked at the study, we did see that the drug caused side effects, which might not meet the researchers’ definition of a “safety concern” but could certainly be of interest to patients taking the drug.
Readers can infer that there was a control group in this study, but that is not clearly stated. We’re also told of at least one important limitation to the results–that the “the study is too small to prove if lampalizumab really helps maintain vision.”
We wish the story had explained that the results are from a phase 2 clinical trial. Phase 2 trials use small samples and looser criteria for effectiveness in order to provide “proof-of-concept” to justify larger and more demanding phase 3 trials. Furthermore, the small sample size used in this phase of research means the margins of error (or confidence intervals) surrounding results are quite large. The original study states that the confidence interval for the 44% figure is 15 to 73%. In other words, the decreased atrophy could actually be anywhere from 15% to 73%. That’s a wide range of possibilities that will only be narrowed with additional research and larger samples.
There is no disease mongering in the article. Macular degeneration is a debilitating and irreversible condition.
Although there are independent sources in the story, we’re not told that one of them–Dr. Rahul N. Khurana–has served as a consultant and speaker for Genentech, the drugmaker behind the study (as disclosed here).
In the story, he’s quoted several times, including here:
“It’s a very, very exciting study,” said Khurana, the ophthalmologist association’s spokesman, who also wasn’t part of the research. “From the basic science perspective, it makes a lot of sense.”
There are currently no approved or effective treatments for the geographic atrophy type of advanced macular degeneration, so there are no known alternatives with which to compare. The story makes this clear.
The story implies that the drug is not yet available, when it explains that the drug company plans to “open two large-scale studies that aim to prove if the drug works. Results are expected later this year.”
The story claims this is the first treatment found to be effective for geographic atrophy macular degeneration. This claim appears accurate, so the novelty of the drug is appropriately established.
With the two outside experts quoted, the story does not appear to rely on a news release.