Two Scientists Receive 2024 Nobel Prize for Discovery of MicroRNA

The Nobel Assembly awarded the 2024 Nobel Prize in Physiology or Medicine to Victor Ambros, PhD, professor of program in molecular medicine at the University of Massachusetts Chan Medical School, and Gary Ruvkun, PhD, professor of genetics at Harvard Medical School, for the discovery of microRNA and its role in post-transcriptional gene regulation.¹

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Ambros and Ruvkun investigated how different cell types develop considering that every cell contains the same set of genes, thus having the same set of genetic instructions. MicroRNAs have played an important role in how organisms develop and function, becoming a new principle for gene regulation. After publication of the results, the scientific community found it interesting, but likely irrelevant to humans and complex animals, according to the Nobel Prize news release. However, in the following years, the article sparked interest, causing hundreds of different microRNAs to be found. Fast forwarding to 2024 and there have now been more than 1000 genes for various microRNA discovered in humans alone. Furthermore, gene regulation by microRNA is now known to be universal among multicellular organisms.¹

Through the findings, scientists have now concluded that abnormal regulation of the microRNAs can contribute to cancer as well as other conditions, including congenital hearing loss and eye and skeletal disorders. Because of this discovery, microRNA used as a potential diagnostic tool for various cancers and conditions as well as finding targets for potential therapeutics.¹

In an interview, Thomas Nickolas, MD, nephrologist and associate professor of medicine at Columbia University, discussed the role of microRNAs in understanding the pathogenesis of renal osteodystrophy (ROD) and how microRNAs can lead to drug development and potential targets for pharmaceuticals.²

“One interesting thing about microRNAs and transcriptomics is that these clue us in better into the underlying pathogenesis of ROD,” Nickolas said. “They also are potentially, not just disease monitoring tools, but also potentially druggable targets. W can use, let's say, microRNAs that are associated with different types of ROD; we can manage them and treat them that will both affect the disease, and then we can use those same microRNAs to monitor therapy similar to what we do for lipids with cardiovascular risk or even albuminuria with [chronic kidney disease] progression.”²

Additionally, the identification of new signatures of microRNAs in August 2023 showed relevance to diffuse large B-cell lymphoma that showed the potential to improve diagnostics, subtype characterizations, and improve treatment response. Further, other results showed the relevance of improving diagnostic approaches and treatment of Clostridioides difficile infections, known to be resistant to antibiotics, and regulating the efficacy of chemotherapy for melanoma.^3-5

Through this discovery, medical professionals and researchers in medicine can now better understand the mechanisms behind microRNA, thus improving therapeutic development for patients.

REFERENCES

1. The Nobel Prize. October 7, 2024. Accessed October 8, 2024. https://www.nobelprize.org/prizes/medicine/2024/press-release/

2. McGovern G. Novel Biomarkers, Diagnostic Approaches for Subtyping Bone Turnover, Improving Patient Outcomes. Pharmacy Times. November 5, 2023. Accessed October 8, 2024. https://www.pharmacytimes.com/view/novel-biomarkers-diagnostic-approaches-for-subtyping-bone-turnover-improving-patient-outcomes

3. Halpern L. MicroRNA Can Assist with Diagnosis, Classification, Prognosis of Diffuse Large B-Cell Lymphoma. Pharmacy Times. August 2, 2023. Accessed October 8, 2024. https://www.pharmacytimes.com/view/microrna-can-assist-with-diagnosis-classification-prognosis-of-diffuse-large-b-cell-lymphoma

4. Halpern L. New, Accurate Detection Methods Necessary for Treating C. Difficile Infections. Pharmacy Times. July 13, 2023. Accessed October 8, 2024. https://www.pharmacytimes.com/view/new-accurate-detection-methods-necessary-for-treating-c-difficile-infections

5. Hunter E. Modulating Levels of miR 204-5p Could Become a Therapeutic Approach for Melanoma. Pharmacy Times. June 28, 2023. Accessed October 8, 2024. https://www.pharmacytimes.com/view/modulating-levels-of-mir-204-5p-could-become-a-therapeutic-approach-for-melanoma