The Hedgehog Review

The Hedgehog Review: Vol. 19 No. 2 (Summer 2017)

Big Promises, Unfulfilled Expectations

Joseph E. Davis

The Hedgehog Review

The Hedgehog Review: Summer 2017

(Volume 19 | Issue 2)

One Team’s Relentless Hunt for a Cure” runs the headline in UVA Today. The article that follows announces that “UVA researchers are on track to eradicate Type 1 diabetes,” while a piece of clever graphic art features the word diabetes with the letters, written in sugar crystals, being blown away.

University of Virginia researchers are “leading the way to a cure”? I had no idea. I read on expectantly. According to the article, an interdisciplinary team is working on early detection…that might be promising…they’re working on a smartphone app to regulate insulin delivery…and making some progress…and they’re embarking on a “journey” to “find a cure.” Hmm. What about eradicating diabetes? Not even close. The ability to generate beta cells—the insulin-producing cells in the pancreas that are destroyed or rendered dysfunctional in people with diabetes—is, we learn toward the end, still “largely uncharted territory.”

The UVA researchers’ journey, underwritten by a $17 million gift from the university’s Strategic Investment Fund, will presumably take them into stem cell and gene therapy investigations. These are considered the most promising approaches to beta cell regeneration. And they are the two areas of research that have, in recent decades, stoked the most powerful popular and professional expectations for a flood of new medical discoveries and treatments.

Indeed, it has become increasingly common for journal articles, university press releases, and scientists in the media to tout such work as “game changing,” “robust,” “amazing,” “novel,” and “unprecedented,” to name but a few of the favored adjectives. Last year, recognizing that things had gotten out of hand, the International Society for Stem Cell Research (ISSCR) issued some overdue anti-hype guidelines. “Potential benefits are sometimes exaggerated,” the society wrote, “and the challenges to clinical application and risks are often understated.” It called for researchers to be accurate, restrained, and circumspect in talking about their work, particularly in making “forward-looking statements on inherently uncertain developments.”

The ISSCR worries that the inflated rhetoric around stem cell research “can have tangible impacts on the expectations” of the general public, patients, physicians, and policymakers. There is plenty of evidence that it already has. Hundreds of stem cell clinics now market unapproved and unlicensed procedures. But the funding agencies themselves may be the greatest victims of the hype.

The overselling of possible cures and even the eradication of diseases has yielded vast outlays for genetic and stem cell research. In a recent opinion piece for the Journal of the American Medical Association, physicians Michael J. Joyner, Nigel Paneth, and John P.A. Ioannidis gave a rough idea of just how much. They found that in 2016 alone, $15 billion (of a total $26 billion) of the National Institutes of Health’s extramural funding went to research in “gene, genome, stem cells, or regenerative medicine.” Joyner and colleagues also document the geometric rise in recent decades in the number of published journal articles on these topics. Scientists are following the money, “recognizing,” the authors say, “that these topics constitute a major locus of important science, financial support, recognition, and prospects for a successful career.”

With so much invested in these areas of biomedicine, it seems fair to ask what the research has accomplished. Joyner and colleagues present an unpromising picture of “underperforming big ideas.” They report that “expectations that a few DNA variants explain most common diseases have faded as the genetic architecture of most diseases has proved to be formidably complex.” It appears that “hundreds or even tens of thousands of genetic variants are involved in each common disease.” But even in single-gene disorders, such as sickle cell anemia, they observe, the biological processes are complex, and knowing the molecular defect does not mean we can target a therapy to address it. “The complex and adaptive nature of most tumors,” they write, “thwarts the optimistic projections for molecularly targeted therapy for cancer.”

The authors also dash cold water on the “prospects of effective treatment based on stem cells.” They give the example of congestive heart failure: “Improvements in cardiac function have been observed only in industry-sponsored studies,” and the only positive relationship between the use of bone marrow stem cells and improvement in cardiac function has been reported in studies with experimental design flaws.

Even electronic health records (EHRs), the big idea that was supposed to employ Big Data to advance “personalized” or “precision” medicine, have not performed as hoped. The Centers for Medicare & Medicaid Services have laid out some $34 billion to incentivize the implementation of EHRs. Yet according to Joyner and his coauthors, the “financial and clinical benefits predicted from shifting to EHRs have also largely failed to materialize.” Inaccuracy, cost overruns, privacy concerns, and other difficulties “make the use of EHRs for research into the origins of disease, as proposed in the Precision Medicine Initiative [a federal research effort] highly problematic.”

There have, of course, been substantial—if now diminishing—reductions in population mortality and morbidity in recent decades. But these gains, Joyner and colleagues argue, have “largely reflected improvement in nonmedical aspects of everyday life and the operation of broad-based public health and classic prevention efforts, such as curtailing smoking,” measures “that are undervalued as outmoded and old-fashioned” in the big ideas regime.

Joyner and colleagues call for a course correction. But while they focus on the direction of biomedical research funding, a real correction would require a larger re-balancing of support for two fundamental orientations in health care: the reductionist and the holistic, or what I elsewhere have called “fixing” and “healing.”

Rather than continue to overfund and overemphasize the promised magic-bullet solutions of biomedicine, the wiser path would be to move toward more innovative and open-ended research (without “specific deliverables”) and away from underperforming initiatives. This more holistic approach would take us not only toward more imaginative and less repetitive biomedical research but also toward work of clear public-health significance.

A greater emphasis on public health requires more than a re-evaluation of funding priorities. It calls for a wider reconsideration of our hopes for therapeutic intervention and a deeper understanding of the “old-fashioned” social and environmental determinants of health and illness.

It won’t be easy for social and public health approaches to compete with big promises for cures, promises that are effectively “future proof,” at least in the intermediate term. For those UVA researchers, won’t every small act of charting the “uncharted territory” of beta cell regeneration constitute progress, suggesting that they are “on track” toward something? Still, the big promises, supported by big money, have created big unmet expectations. Perhaps the time is coming for a reset.

Reprinted from The Hedgehog Review 19.2 (Summer 2017). This essay may not be resold, reprinted, or redistributed for compensation of any kind without prior written permission. Please contact The Hedgehog Review for further details.

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Published three times a year by the Institute for Advanced Studies in Culture, The Hedgehog Review offers critical reflections on contemporary culture—how we shape it, and how it shapes us.

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