Just in time for the annual awards of the Nobel prizes, Stat, the online health news site, provides a trenchant reminder that science advances slowly. That’s especially true in medical science. So as international attention turns to the announcements from Stockholm starting Monday, it’s important to contain the hype and keep in perspective how lauded research does, or does not, move into wider application.
It was only a decade ago, and just eight years after they published their pioneering research on a basic, if yuck-inducing, form of life─the parasitic roundworm─that two academics found themselves thrust into an international spotlight, Stat says.
They were hailed as scientific geniuses, and their work launched a gold rush by Big Pharma to exploit the promise of their pioneering work on RNA interference. So-called RNAi, the conventional wisdom held, would provide big breakthroughs in fighting an array of diseases and conditions, because, at least in the lowly roundworm, the two Nobel Laureates had found that select genes could be shut off, meaning their harmful effects could be readily erased.
Billions of dollars of Big Pharma cash flooded in; researchers stampeded into the sector.
But the quick win failed to materialize. Processes that worked in a simple intestinal bug seemed to replicate in significantly more complex mammals. They turned out badly, though, in people, with some testing of early RNAi drugs flopping and causing bad side-effects in patients. It became clear that one of the huge challenges also would be how to deliver RNAi medications to the exact cells where they would help.
Over the years, the two innovating scientists found themselves increasingly lonely in their difficult work, as the money, research colleagues, and dazzle drained away. One Big Pharma outfit poured $1 billion into a firm to develop RNAi drugs; it eventually let the company go for $175 million. It was bought up, in turn, by another company that committed to the long term and plunked $1 billion more for further research.
And now, that firm is expressing confidence that late-stage human trials will finally green light an RNAi drug “for a rare, life-threatening disease that results from the buildup of misfolded proteins,” Stat says.
Patience is critical when pursuing medical science research, especially when initial, promising efforts achieve as great an honor as the Nobel, the site reports. It deserves kudos for this observation (with which I could not more heartily agree), as well as a recent piece it posted with the headline, “Beware the hype: Top scientists cautious about fighting cancer with immunotherapy.”
That story cut against the conventional wisdom that there will be speedy “breakthroughs” in cancer care with therapies that boost the body’s own abilities to battle disease. Stat notes that careful scientists fret not only about the froth surrounding cancer immunotherapy, but also about the hype for optogenetics (light-based cell control) and CRISPR-Cas9 (a promising tool to “edit” genes).
As I wrote recently, however, researchers have much work to do figuring out critical aspects of two of the most common and lethal cancers, breast cancers afflicting women and prostate cancer in men. In both instances, evidence-based medicine after long struggles has forced practitioners to reconsider the effectiveness of frequent screenings for all when those tests in themselves might lead to greater harms and not improve cancer care nor reduce its deaths. A new, decade-long study of thousands of men gave greater support to “watchful waiting,” the decision to postpone major, invasive procedures to preserve quality of life and to determine better if a man’s disease was slow-growing and more acceptable to mostly leave be or if it requires aggressive care. Breast cancer researchers also are rethinking surgeries and how extensive these need to be, including whether it is necessary to routinely remove extensive amounts of women’s lymph nodes.
Yes, true scientists deserve every laurel and accolade possible for their difficult, dogged work. But they also should not be turned into nor allow themselves to become part of the circus of hype.
