Nobel Prize in medicine honors 2 scientists for their discovery of microRNA

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STOCKHOLM (AP) — Two scientists won the Nobel Prize in physiology or medicine on Monday for their discovery of microRNA, tiny bits of genetic material that offer a way for scientists to control what’s happening in our cells and that could lead to new ways of detecting and treating diseases including cancer.

The work by Americans Victor Ambros and Gary Ruvkun is “proving to be fundamentally important for how organisms develop and function," according to a panel that awarded the prize in Stockholm.

Ambros and Ruvkun were initially interested in genes that control the timing of different genetic developments, ensuring that cell types develop at the right time.

Their discovery ultimately “revealed a new dimension to gene regulation, essential for all complex life forms,” the panel said.

RNA is best known for carrying instructions for how to make proteins from DNA in the nucleus of the cell to tiny cellular factories that actually build the proteins. MicroRNA does not make proteins, but help to control what cells are doing, including switching on and off critical genes that make proteins.

Ambros and Ruvkun studied two mutant strains of worms commonly used as research models. The scientists set out to identify the mutated genes responsible for cell development in these worms and what their role was. The mechanism they ultimately identified — the regulation of genes by microRNA — has allowed organisms to evolve for hundreds of millions of years.

“Their groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans,” according to the citation explaining the importance of their work.

Ambros, currently a professor of natural science at the University of Massachusetts Medical School, performed the research at Harvard University. Ruvkun’s research was performed at Massachusetts General Hospital and Harvard Medical School, where he’s a professor of genetics.

The study of microRNA has opened up approaches to treating diseases like cancer because it helps regulate how genes work at the cellular level, according to Dr. Claire Fletcher, a lecturer in molecular oncology at Imperial College London.

Fletcher said there were two main areas where microRNA could be helpful: in developing drugs to treat diseases and in serving as possible indicators of diseases, by tracking their levels in the body.

“If we take the example of cancer, we’ll have a particular gene working overtime, it might be mutated and working in overdrive,” said Fletcher. “We can take a microRNA that we know alters the activity of that gene and we can deliver that particular microRNA to cancer cells to stop that mutated gene from having its effect.”

Eric Miska, a geneticist at Cambridge University, said the discovery by Ambros and Ruvkun came as a complete surprise, overturning what scientists had long understood about how cells work.

“It was just a shock that there’s this whole new class of gene, the genes that make these microRNAs that have been missed," he said. He said the human genome has at least 800 microRNAs that are critical to determining how cells function.

Miska said there is ongoing work regarding the role of microRNA in infectious diseases like hepatitis and that it might also be useful in helping treat neurological diseases.

Fletcher said there are research studies ongoing to see how microRNA approaches might help treat skin cancer, but that there aren’t yet any drug treatments approved by drug regulators. She expected that might happen in the next five to 10 years.

She said microRNA represents another way of being able to control the behavior of genes to treat and track various diseases.

“The majority of therapies we have at the moment are targeting proteins in cells,” she said. “If we can intervene at the microRNA level, it opens up a whole new way of us developing medicines and us controlling the activity of genes whose levels might be altered in diseases.”

The phone call from the Nobel panel is often a surprise, but there are certain signs that recipients and their families pick up on.

“Well, when a phone rings at 4:30 in the morning. ... It never happens here. So we answered,” Ruvkun said.

And his wife heard another giveaway. “My wife figured out that it was ... because Swedish accent,” he added.

It took a little longer to rouse Ambros.

“Somebody called my son, who called my wife as my phone was downstairs,” he said.

Ruvkun knew immediately the impact the award would have on his life.

“Well, I just kept repeating in my mind, this changes everything because you know, the Nobel is just mythic in how it transforms the life of people who are selected,” Ruvkun said. “The Nobel Prize is a is a recognition that’s sort of 100 times as much press and celebration as any other award. So, it’s not part of a continuum. It’s a quantum leap.”

Going to pick up his award in December will be the third time he has been to a Nobel Prize ceremony in Stockholm after attending to watch his mentor Robert Horvitz receive the 2002 award and then his buddy Jack Szostak, who won in 2009.

“There’s a trip coming up. It will be the third, possibly the best,” Ruvkun said.

Ambros said he didn’t expect the award as he felt that the Nobel committee has already singled out RNA in the 2006 prize that went to his friends Andrew Fire and Craig Mello.

“It represents the recognition of how wonderful and unexpected discoveries come from a curiosity in basic science financed by taxpayer money. It’s a vitally important, probably the most important message that this investment really pays off,” he said.

Last year, the Nobel Prize in physiology or medicine went to Hungarian-American Katalin Karikó and American Drew Weissman for discoveries that enabled the creation of mRNA vaccines against COVID-19 that were critical in slowing the pandemic.

The prize carries a cash award of $1 million from a bequest left by the prize’s creator, Swedish inventor Alfred Nobel.

Nobel announcements continue with the physics prize on Tuesday, chemistry on Wednesday and literature on Thursday. The Nobel Peace Prize will be announced Friday and the Nobel Memorial Prize in Economic Sciences on Oct. 14.