The 2024 Nobel Prize in Physiology or Medicine has been awarded to Victor Ambros and Gary Ruvkun, key figures in the development of microRNA; miniscule RNA molecules that enable cells to control which proteins they produce.
The Nobel Assembly of Sweden’s Karolinska Institute, which selects the prize’s winner said that “[Ambros and Ruvkin’s] groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans. It is now known that the human genome codes for over 1,000 microRNAs.”
To get to this point, the two professors experimented on a mutant form of a tiny roundworm known as C. elegans which failed to develop certain cell types. They eventually discovered tiny pieces of genetic material – microRNAs – that turned out to be an essential factor in the worms’ development.
Explaining the value of this technology to PharmaBoardroom, Angela Zhou of CAS, a division of the American Chemical Society that specialises in scientific knowledge management, noted that “microRNAs are small molecules produced by our cells that help regulate production of proteins. They act like dials that fine-tune expression of genes through mRNA transcription. microRNAs are essential for the growth, development, and even death of our cells.”
Ambros told Reuters that he characterised microRNA as “a communication network amongst genes that enables the cells in our bodies to generate all kinds of different complex structures and functions.”
As a BBC explainer lays out, a gene, essentially a set of genetic instructions, is embedded in our DNA. Our cells create a copy of this gene, known as messenger RNA (mRNA). The mRNA leaves the cell’s nucleus and directs the cell’s machinery to produce a specific protein. However, microRNAs interfere by attaching to the mRNA, preventing it from functioning. As a result, the gene’s expression is blocked in the cell. Subsequent research revealed that this mechanism is not limited to worms but is in fact a fundamental process across life on Earth.
The discovery of microRNA has been a major breakthrough for the medical and pharmaceutical field. The misfunctioning of microRNAs has been shown to contribute to diseases like cancer, heart issues, Alzheimer’s, congenital hearing loss, bone disorders and diabetes. “Currently, there are multiple drugs being tested in clinical trials for cancer and heart disease that target microRNAs to regulate or mimic critical protein pathways,” adds Zhou.
A broad spectrum of organisations is involved in this effort, with US biotech Alnylam Pharmaceuticals currently one of the leading players, along with the German research institute the Max Planck Society, and another American firm Thermo Fisher Scientific. GlobalData analysis shows that Alynlam has filed the most microRNA patents of any company over the past three years.
Ambros, now 70, of the University of Massachusetts Medical School, and Ruvkun, 72, from Harvard Medical School, will share the 11m Swedish kronor (USD 1.1m) prize fund. They follow in the footsteps of last year’s winners Katalin Kariko and Drew Weissman, who developed the technology that led to mRNA COVID-19 vaccines from the likes of Moderna and Pfizer/BioNTech.
