Although not mere genetic waste, microRNA holds significant potential in the medical field.
For Centuries Upon Centuries, microRNAs Have Influenced Human Health and Welfare. They're Capable of Inflicting Suffering, Yet Also Aid in Alleviating or Even Curing It. There Are Various Methods That Might Not Have Been Conceivable Without the Discovery of This Year's Nobel Prize Winners.
Diseases such as cancer, autoimmune disorders, and cardiovascular issues frequently involve gene regulation. Since the 1960s, messenger RNA (mRNA) was recognized for carrying the proteins' instruction blueprint within cells. However, non-coding RNAs, devoid of such data, were once considered genetic debris. This year's Nobel Laureates, Victor Ambros and Gary Ruvkun, have debunked this misconception for microRNAs (miRNA).
These ambassador molecules guided organism growth and subsequently human health for countless millennia. Manufactured by body cells, miRNAs ensure the production of specific proteins at the appropriate time, location, and volume in the proper body tissue.
However, miRNAs also play a part in disease processes. Tumors, heart failure, inherited hearing loss, and visual impairments are examples. Single protein mutations required for miRNAs can cause DICER1 syndrome, a rare condition that triggers tumors in various organs. Contrariwise, miRNAs can also be utilized as disease antagonists - either by inhibiting them or introducing synthetic alternatives.
Preventing Heart Failure?
Though no approved treatments exist 30 years after Ambros and Ruvkun's discovery in the roundworm, various applications are now on the verge of medical implementation: Human studies examine whether miRNAs can be utilized for disease diagnosis or therapy.
Heart Failure Example
Heart failure occurs when the heart is too weak to pump blood throughout the body. Over 40,000 Germans succumb to this disease annually, as per the Heart Foundation. Hanover-developed drug CDR132L allegedly binds to microRNA-132, contributing to heart muscle scarring - it aims to slow down or even halt heart failure progression.
"Our team was the first to demonstrate in 'Nature' in 2008 that microRNA can be therapeutically employed against heart failure in mice," explained Thomas Thum of the Hannover Medical School (MHH). "Since then, we have undertaken the world's largest Phase 2 study with almost 300 heart attack patients using a microRNA strategy." Thum, Cardior's founder, anticipates "a new generation of miRNA-based medications" suitable for various diseases. Research is also being carried out for kidney and lung fibrosis.
Detecting Alzheimer's Earlier in a Single Drop of Blood
Cancer Example
miRNA plays a significant role in tumors, according to Sven Diederichs of the German Consortium for Translational Cancer Research at the University of Freiburg. miRNAs may suppress tumor-suppressing genes, and their loss can activate cancer genes.
miRNAs can be employed diagnostically due to their distinctive tissue traits. In pancreatic and lung cancer, this could not only aid early detection but monitor therapeutic progression. miRNA-based therapies are currently being tested - not just in pancreatic tumors but also in lung and brain cancer.
Alzheimer's Example
miRNAs can improve early detection of the most prevalent form of dementia, as an international research team claims in "Alzheimer's & Dementia" following a study of 800 individuals. André Fischer from Göttingen University Hospital stressed the importance of therapies for Alzheimer's, but also the necessity for new methods to identify the disease prior to symptomatic memory loss. "We have discovered that this is possible through a measurement of microRNAs in the blood," Fischer said.
The procedure has yet to be designed for clinical application. They're working on a simple blood test that can detect several miRNAs from a single droplet of blood. Fischer believes this test could be available within a few years. It could supplement more intricate procedures such as cerebrospinal fluid examination and brain scans.
However, no miRNA-based medications have been approved. Considering that these RNA constituents have only been acknowledged in humans for two decades, the research's progress is remarkable, stated Diederichs. The fact that this approach has therapeutic potential is also indicated by an economic acceleration: In May, the Danish pharmaceutical company Novo Nordisk - renowned for the weight-loss injection Wegovy - acquired Cardior, founded by Thum, for approximately one billion euros.
MiRNAs, discovered to play a crucial role in cancer development, can potentially aid in early diagnosis and monitoring of treatment progression. For instance, in pancreatic and lung cancer, miRNAs can distinguish between healthy and cancerous tissue, enabling early detection and improved prognosis. Similarly, miRNA-based therapies are currently being tested for various types of cancer, including pancreatic, lung, and brain cancer.
