SMART NEWS

American Scientists Win Nobel Prize in Medicine for ‘Groundbreaking’ Gene Discovery Made by Studying Worms

Victor Ambros and Gary Ruvkun discovered microRNA, tiny molecules that play a crucial role in how cells develop, paving the way for new treatments for diseases

Alexa Robles-Gil

Daily Correspondent

a journalist takes a picture with a phone of the two Nobel winners projected on a screen in a lecture hall
The Nobel Prize in Physiology or Medicine was awarded to Victor Ambros and Gary Ruvkun, who together discovered microRNA. Jonathan Nackstrand / AFP via Getty Images

On Monday, American scientists Victor Ambros and Gary Ruvkun won the Nobel Prize in Physiology or Medicine for their “groundbreaking” discovery of microRNA, tiny molecules that are important for gene expression. Ambros and Ruvkun worked together as postdoctoral researchers and first published their discoveries in 1993.

The two scientists, who will share the prize of 11 million Swedish kronor (around $1 million), were both excited to win the esteemed award together. Ambros says he is “particularly happy to share this with Gary Ruvkun, who’s a great friend of mine,” to Reuters’ Jonathan Allen.

For his part, Ruvkun was cautiously delighted when he heard he won the prize—as the idea that the phone call was a prank played at the back of his mind. “The call from Stockholm is mythic in the world of science,” he says in a phone interview with the Nobel Committee. Besides the initial surprise, his first thoughts were, “oh boy, it’s going to be a fun ride!”

Ambros, who is a researcher at the University of Massachusetts Medical School, and Ruvkun, a geneticist at Harvard Medical School, helped explain how cells specialize and develop into various types, such as muscle cells or nerve cells. They revealed “a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans,” according to a statement from the Nobel Committee. Now, their work is providing the basis for treating diseases such as cancer.

The two Nobel laureates started working together in the late 1980s as postdoctoral fellows in the lab of Robert Horvitz, who became a Nobel laureate himself in 2002. They studied a small roundworm called Caenorhabditis elegans, which is commonly used as a research model in biology. The pair examined mutant genes in this roundworm, trying to better understand their function.

Through that research, they uncovered an unusually short RNA molecule—called microRNA—that could bind to messenger RNA (mRNA) and control how much protein it produces. That finding revealed a new way that cells regulate which genes are expressed.  

Their findings were published in 1993 in two separate papers in the journal Cell. At first, it was thought the discovery was unique to roundworms, and the scientists’ work was met with “almost deafening silence,” the Nobel Committee says in the statement. But in 2000, Ruvkun’s research team published another finding in Nature that showed microRNAs were present in other animals as well.

Today, scientists know the human genome codes for more than 1,000 different microRNAs.

“It’s a completely new physiological mechanism that no one expected, completely out of the blue, and it shows that curiosity research is very important,” Olle Kämpe, a member of the Nobel Committee, said after the prize announcement, per Science’s Catherine Offord.

After the discovery of microRNA, scientists learned that the tiny molecules are crucial to how cells and tissues develop. Mutations in the genes that code for microRNAs can lead to eye and skeletal disorders, as well as congenital hearing loss. And abnormal microRNA can cause too much or too little of a protein, which can contribute to cancer.

Such findings “opened up a whole new understanding of how diseases happen, which means that we have new possibilities for reversing them,” Jon Lorsch, an institute director at the National Institutes of Health, tells the New York Times’ Teddy Rosenbluth and Derrick Bryson Taylor.

The study of microRNA and gene regulation could be useful for developing drugs or indicating the presence of disease, as Claire Fletcher, a molecular oncologist at Imperial College London, says to the Associated Press (AP).

“If we take the example of cancer, we’ll have a particular gene working overtime, it might be mutated and working in overdrive,” Fletcher tells the AP. Eventually, these effects might be stopped using microRNA, she adds.

RNA has been involved in at least five Nobel Prizes, including last year’s physiology and medicine prize, which went to scientists who paved the way for mRNA vaccines. And this year, the Nobel Committee points out, the award-winning RNA research started with scientists’ curiosity about a small worm.

“It’s striking that the work on the simple worm C. elegans is still resulting in Nobel Prizes,” Ronald Plasterk, a Dutch politician and molecular biologist who previously worked on microRNA, tells Science. “So much basic biology has been discovered in this simple animal of only 959 cells.”

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Alexa Robles-Gil

Alexa Robles-Gil is a bilingual science journalist based in New York City. Her work has appeared in the Guardian, Undark, Inside Climate News and more.