Nobel Prize for medicine recognizes microRNA — and curiosity

In a climate where so much time and energy is spent trying to tackle human disease, Monday’s Nobel Prize in the category of physiology or medicine is a welcome reminder of the value of pursuing research that scratches a scientific itch. Sometimes, that work might lead to a new understanding of human biology.

“Curiosity research is very important,” said Olle Kämpe, member of the Nobel Committee for Physiology or Medicine, in announcing the prize to Victor Ambros, a professor at the University of Massachusetts Medical School, and Gary Ruvkun, at Harvard Medical School, for discovering so-called microRNA. MicroRNA is one of a handful of biological systems that carefully orchestrate when, where and how intensely DNA should operate.

“They were looking at two worms that looked a bit funny and decided to understand why. And then they discovered an entirely new mechanism for gene regulation. I think that’s beautiful,” Kämpe said.

I do, too.

The fundamental discoveries underpinning today’s award were conducted in a lowly roundworm. C elegans is a millimeter-long organism made up of a manageable 1,000 cells (Humans have somewhere between 28 and 36 trillion) yet also boasts a surprising sophistication that has made it a workhorse in research labs. In fact, this is the third set of scientists that can thank the organism for their Nobel nod.

Studying the roundworm allowed Ambros and Ruvkun to add needed nuance to biology’s central dogma that DNA is copied to RNA, which then is translated into proteins inside cells. But that simple, linear story can’t address a fundamental question: If each of our trillions of cells carry the exact same genetic blueprint, how can a neuron look and behave so differently from, say, heart tissue or a muscle cell? As the researchers helped explain, it’s all about when various proteins are turned on and off — a process that these small stretches of RNA play a vital role in regulating.

Understanding this deeper layer of gene regulation took decades. After Ambros described the first microRNA in 1993, it was seven more years before Ruvkun unveiled the second one. Soon it became clear that humans, too, shared these molecules and the field exploded as researchers identified the snippets of code in various organisms.

While microRNA’s central role in regulating gene expression hasn’t yet directly led to approved treatments or a groundbreaking vaccine, that doesn’t mean that microRNA won’t one day have an impact on our health. The discovery has already been vital to our knowledge of human disease.

MicroRNAs can tamp down expression of genes that allow healthy cells to go through their normal process of dying and cause the kind of uncontrolled cell growth that is a hallmark of cancer, explains Matthew Disney, a chemistry professor at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology. Already there is compelling evidence that microRNAs are misbehaving in cancers such as so-called triple-negative breast cancer and brain cancer.

And while turning microRNA into a diagnostic or targeting it with a medicine is tricky, academic scientists and biotech companies are hard at work on it. Disney is the founder of one of several biotech companies devoted to tapping into the therapeutic potential of harnessing microRNA. Given that microRNA levels are out of whack in so many diseases, others are working on ways to use the tiny molecules as a diagnostic tool.

The discovery of microRNA also emphasizes the importance of all the dark matter in our genome. Of the several billion base pairs that make up a human genome, only a small portion make up the recipes for proteins. So what is the rest of that so-called “non-coding” material for? Over the past few decades, scientists have started to understand that it’s far from junk — rather, buried within is the code for vital molecules, like microRNA.

We can’t lose sight of the value of these kinds of astonishing, foundational discoveries. “Our government has gone more and more towards funding the type of research that is only connected to a disease,” says chemist Thomas Cech, who won the Nobel Prize in chemistry in 1989 for a discovery that helped illuminate RNA’s essential role in life. And yet these foundational breakthroughs in biology are what lay the groundwork for better understanding human health.

This prize underscores the need to strike the right balance between basic research and direct applications to medicine. As Ambrose and Ruvkun’s work makes clear, no matter how much we think we know about our inner workings, biology has a way of reminding us how much left there is to discover.

This column does not necessarily reflect the opinion of the editorial board or Bloomberg LP and its owners. Lisa Jarvis is a Bloomberg Opinion columnist covering biotech, health care and the pharmaceutical industry. Previously, she was executive editor of Chemical & Engineering News.