Victor Ambros and Gary Ruvkun win Nobel Prize in Medicine for microRNA gene breakthrough

US scientists recognised for physiology research involving tiny worms

Nobel committee secretary general Thomas Perlmann announces the winners of the 2024 Nobel Prize in Physiology or Medicine. AFP

The Nobel Prize in Medicine has been awarded to the Americans Victor Ambros and Gary Ruvkun for their discovery of microRNA, a fundamental principle governing how gene activity is regulated which could lead to new ways of treating cancer.

The committee said their discovery is “proving to be fundamentally important for how organisms develop and function”.

Their research was based around the study of the make-up of a one millimetre-long roundworm, known as C.elegans, which despite being tiny possesses cell types such as nerves and muscles found in larger, more complex animals. Understanding the regulation of gene activity has been an important goal for decades, the jury said. If gene regulation goes awry, it can lead to serious diseases such as cancer, diabetes, or autoimmunity.

"Their groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans," the jury said. Mr Ambros, 70, performed the research that led to the pair winning the Nobel Prize in 1993 at Harvard University. He is currently a professor of natural science at the University of Massachusetts Medical School.

Seven years later Mr Ruvkun’s research was performed at Massachusetts General Hospital and Harvard Medical School, where he is a professor of genetics, said Thomas Perlmann, secretary general of the Nobel committee. "I was able to wake up Gary Ruvken," Mr Perlmann said. "He was very excited and happy. He was so enthusiastic and thrilled to be coming to Stockholm."

The groundbreaking findings on gene regulation, which allows each cell to select only relevant instructions, was first published in two articles in 1993. The jury said many trials into the treatments of cancer and kidney problems that use the research are currently in progress.

"The impact of understanding the basic functions is always the first step towards using this knowledge," they said. The committee's vice chair, Professor Olle Kämpe, said some tumours delete and mutate microRNAs, and it is hoped this knowledge will help in the search for treatment.

Dr Claire Fletcher, a lecturer in molecular oncology at Imperial College London, said microRNA provide genetic instructions to tell cells to make new proteins, and that there were two main areas where microRNA could be helpful: in developing drugs to treat diseases and in serving as biomarkers.

“MicroRNA alters how genes in the cell work,” she said. “If we take the example of cancer, we’ll have a particular gene working overtime, it might be mutated and working in overdrive. 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.

"The majority of therapies we have at the moment are targeting proteins in cells. 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.”

Last year, the Nobel Prize in Physiology or Medicine went to the Hungarian-American Katalin Karikó and the American Drew Weissman for discoveries that enabled the creation of mRNA Covid-19 vaccines, which were vital in slowing the pandemic.

The prize carries a cash award of 11 million Swedish kronor ($1 million) from a bequest left by its creator, the Swedish inventor Alfred Nobel. The announcement launched this year’s Nobel prizes award season.

Nobel announcements continue with the physics prize on Tuesday, chemistry on Wednesday and literature on Thursday. The Nobel Peace Prize will be announced on Friday and the Nobel Memorial Prize in Economic Sciences on October 14. The laureates are invited to receive their awards at ceremonies on December 10th, the anniversary of Alfred Nobel’s death.