‘Pay attention to revolutions’: Mass. scientists win Nobel Prize in Medicine for discovery of microRNA

Two scientists from Massachusetts are this year’s winners of the Nobel Prize in physiology or medicine.
 
Victor Ambros of UMass Medical School and Gary Ruvkun of Harvard Medical School and Massachusetts General Hospital won for their discovery of microRNA — tiny bits of genetic material that serve as on and off switches that help control what cells do.
 
Ambros said at a press conference that he was sleeping in this morning and missed a few calls. He finally found out about the award when his son reached out with an important message.

“‘If you get a call from somebody in in Sweden, answer it!’” Ambros recalled his son saying. “They had called him. So I guess the poor people over there were getting desperate and they started [making phone calls], ‘All right, anybody Ambros in the general area,’ you know?“

At a separate press conference at MGH, Ruvkun commented that a Nobel was unlike any award he’s gotten before.

”The Nobel is is its own class in terms of how much attention it gets,” he said. “I’ve probably gotten 10 different awards over the last 20 years. There’s never been a press conference like this for TV cameras. Nothing like this. It’s a completely different world.”

Ruvkun was asked by a 25-year-old scientist in the room what advice he’d give his 25-year-old self, and described an inauspicious beginning to his career.

“From, like 21 to 24, I was a disappointment to anybody who was thinking about what my where my career might go,” he said. “You know, because I lived in my van and planted trees in the Pacific Northwest and traveled in third-class buses to Tierra del Fuego for a year.”

Ruvkun recounted things changing in his life when he encountered a stack of Scientific American magazines while traveling in Bolivia.

“And I spent the day reading Scientific American, and it was really a good day,” he remembered. “And I said, ‘You know, maybe I’ll go to graduate school.’”

Ruvkun said he went to Harvard in 1976, just as the study of recombinant DNA was starting to take off, and he said at that moment the area of study was a revolution — one that reminded him of quantum physics revolution that he studied as an undergrad.

“Advice to a 25-year-old?” he said, returning to the question. “Pay attention to revolutions. And enjoy them, because they don’t happen that often. And they’re works of art.”

At his celebratory press conference at UMass Chan Medical School in Worcester, Ambros wore a short-sleeved shirt decorated with tigers that he said his wife and scientific collaborator, Rosalind “Candy” Lee, had bought for him at a secondhand store.

He thought back on the decades and even centuries of moments that led to this momentous honor.

“All of us in this room, I think, can probably say to ourselves, ‘Why? How come I’m here?’ Think about my grandparents, the journeys my parents took, you know, and people before that. I’m a son of an immigrant from Poland. Candy’s a daughter of immigrants from China. And in these stories, there’s heroes that you hear about in our history that are everyday heroes who brought us here.”

Ambros said his father was a refugee from Poland with a limited education, a man who was captured by the Nazis and worked as a slave laborer for years. He said his father was later liberated and befriended a U.S. soldier, who sponsored his immigration to the United States.

“And so that’s how I’m here,” he said.

His Nobel-winning research began with a question about a genetic mutation in a worm, one that caused it to transition to later developmental stages abnormally quickly.

“So the animal would reach an adult stage with lots of adult parts missing, because it never made those parts, and it had extra parts that are produced in the early larval stage,” he said. “So I think you can imagine that this would be a really interesting mutant to study.”

The responsible mutation they ultimately found broke the sequences where a small RNA was being produced.

“It was the first microRNA to be identified,” Ambros said. “It’s called a ‘micro RNA’ because it’s really unexpectedly short. It was surprising that there could be enough information contained in only 22 bases — or code words — in the genome for this microRNAs to regulate another gene so precisely.”

Ambros recalled meeting families of children who have a genetic disorder related to microRNA called Argonaute Syndrome that causes developmental delays.

“So the basic scientists in the audience were learning from the mutations that were presented to us by the families,” he said. “And several families were there at the meeting and several of the children were there and we got to meet them. And for me, this was an amazing experience because I was, for the first time, trying to process this idea that we basic scientists are learning from these patients.”

The study of microRNA has opened up approaches to treating diseases like cancer because it helps regulate how genes work in our cells, said 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 microRNA 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. She said scientists might one day be able to use microRNA to stop such effects.

Ambros said the processes that manage our cells are still mysterious, despite advances in understanding gene sequences.
 
“At any given moment, it feels like we know most of what we need to know,” he said. “But that is actually an illusion that we have to consciously disabuse of ourselves of and leave ourselves open for the surprises.”

Embracing those scientific “surprises” was also an important theme for Ruvkun.

“The surprises are what keep you young in science,” Ruvkun said. “And so I am constantly surprised. And my ignorance is bliss.”

This story uses material from the Associated Press.