Everyone, it seems, knows about the Ice Bucket Challenge, the viral phenomenon that raised record-breaking sums for the Amyotrophic Lateral Sclerosis (ALS) Association. This feel-good movement was critiqued by many, but no one can claim it was a bad thing: It raised lots of money, the overwhelming majority (96 percent) of which was used to support research or services or education related to this horrible disease. What’s wrong with that?
The problem arises when we indulge the false narrative that these sorts of short-term infusions of money lead to major scientific “breakthroughs.” This absurd argument — that the Ice Bucket Challenge has led to a “breakthrough” in ALS research — is now being made on the pages of the venerable Washington Post, and was widely picked up by many other news outlets.
The reporting was based on a study that first appeared in Science magazine, which explained issues relating to a dysfunctional protein in ALS patients, suggesting that this discovery could lead to new therapeutic strategies in treating the disease.
Here’s why I take issue with much of the reporting on this research.
1. This new finding is not a “breakthrough.” I say this because, intrigued by the hype, I went back to read the original paper on which this story was based. Okay, full disclosure: I really tried to read the paper, but it’s full of biological mechanisms and scientific jargon that I don’t fully understand, so I can’t say I read (or absorbed) it in its entirety. (Feel free to read the paper in Science yourself; perhaps you’ll do better than I did.) Still, I understood it sufficiently to conclude that this study is elegantly conducted and yields important scientific insights. However, it is not a breakthrough — at least, not in the sense that people who suffer from an illness would use the term — because:
- The study was done on mice and HeLa cells, not patients. Such studies may enhance our understanding of diseases and potential treatments — as this one almost certainly does — but only occasionally lead to clinical applications, and if then, usually after many years.
- The authors’ conclusions in the published paper (though not in their postpublication video; more on that later) are reasoned and limited, and not even vaguely breakthrough-ish. “Furthermore, repression of cryptic exons was impaired in ALS-FTD cases, suggesting that this splicing defect could potentially underlie TDP-43 proteinopathy.” Maybe they are suggesting this could potentially be a breakthrough (someday)?
- The press release from Johns Hopkins doesn’t use the word “breakthrough” (nor does it read like they thought of it as one).
And let me be clear: By saying it’s not a breakthrough, I’m not saying it’s not important and potentially useful. I’m only objecting to the word “breakthrough,” which has specific implications to the general public.
2. This study wasn’t made possible by the Ice Bucket Challenge. In their YouTube video, the lead author inappropriately describes this as “a big breakthrough for ALS.” (Which he clarified later by saying: “So with any luck, this could lead to possibly a cure or at least really slowing down this terrible disease.”) However, the senior scientist points out that this builds upon 20 years of work. Moreover, the acknowledgments in the paper note six funding sources other than the ALS Association (ALSA) (including the NIH). The total amount from ALSA was $160,000. I dare say the several other sources of support that were also listed would have allowed this “breakthrough” even without ALSA’s relatively small contribution.
3. Hyping research findings takes unfair advantage of those in dire straits. Overplaying the significance of research findings undermines the public’s confidence in science. It is also painfully common: for instance, of 101 basic science studies in top-tier scientific journals that promised major clinical applications, only five led to approved treatments 20 years later. This epidemic of hype has many underlying causes, including incentives for the scientists and the institutions they work for, but it is dangerous and must be contained.
4. You can’t solve a major scientific/clinical problem simply by throwing money at it. I am of an age that I remember Nixon’s “War on Cancer.” (I was in high school in 1971, when that war was declared.) Need I say more?
5. It’s OK to use marketing techniques to sell laundry detergent, but not to drive scientific priorities. It has been pointed out that the Ice Bucket Challenge put disproportionate attention (and funding) into a rare disease. Now, I’m not saying that research funding should be directly proportional to the number of people afflicted with or dying from an illness, but at least there ought to be some scientific or policy rationale for how limited research dollars are allocated. Pulling on the heartstrings, or bringing in celebrities, should not be the way these decisions are made. Besides, ALS has already got the marketing advantage of a great, celebrity-studded moniker in “Lou Gehrig’s disease,” something that other rare diseases, I’m sure, would love to have. Wouldn’t it be nice to have some logical way to allocate research funding where it will serve the greatest good?
Please understand: I certainly hope that ALS will be cured someday, and perhaps this new mechanistic insight will contribute to that outcome. But let us not mislead the public about the significance of individual, incremental steps in the arc of science, nor promulgate the notion that “crowdsourcing” scientific discovery will get us where we need to go.
Paul Marantz is associate dean, clinical research education and director, Center for Public Health Sciences, Albert Einstein College of Medicine, Bronx, NY. He blogs at the Doctor’s Tablet.
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