The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (miRNA) and its fundamental role in gene regulation. This breakthrough has revolutionized the understanding of how genes function at the cellular level and opened potential new avenues for treating diseases, particularly cancer.
The Nobel Assembly at the Karolinska Institute recognized that Ambros and Ruvkun's work has proven to be fundamentally important in understanding how organisms develop and function. Their research, conducted independently but collaboratively, focused on the roundworm C. elegans to investigate cell mutations and their timing. This led to the identification of microRNAs, a novel class of small RNA molecules that regulate gene expression by selectively instructing cells.
"The seminal discovery of microRNA has introduced a new and unexpected mechanism of gene regulation," said Thomas Perlmann, Secretary-General of the Nobel Assembly. He added that microRNAs are crucial for understanding embryological development, normal cell physiology, and diseases such as cancer.
MicroRNAs function by altering how genes in the cell operate. Dr. Claire Fletcher, a lecturer in molecular oncology at Imperial College London, explained that in diseases like cancer, where a particular gene might be overactive or mutated, microRNAs can be delivered to cancer cells to halt the mutated gene's effect. This offers a new approach to drug development and biomarker discovery.
Currently, clinical trials are underway to explore the potential of microRNA-based therapies, particularly in treating skin cancer, and also cardiovascular and kidney diseases. While no microRNA-based drug treatments have yet been approved by drug regulators, experts anticipate potential approvals within the next five to ten years.
Gary Ruvkun, a professor of genetics at Harvard Medical School, expressed his excitement upon receiving the news, stating, "It's quite a sea change." Victor Ambros, a professor of natural science at the University of Massachusetts Medical School, also shared his astonishment and delight.
The Nobel Prize includes a cash award of 11 million Swedish kronor ($1 million), a diploma, and a gold medal. The laureates will receive their awards at a ceremony on December 10 in Stockholm.
