Katalin Karikó on mRNA's Next Frontier: Beyond COVID

Medscape

5 hours ago

There is the aloof scientist, locked in an ivory tower, decidedly uninterested in (and perhaps ill-suited to) engaging with 'ordinary people.' And then there's Katalin Karikó, who, along with Drew Weissman, won the 2023 Nobel Prize in Physiology or Medicine. Karikó is a person of extraordinary kindness and openness who introduces herself to researchers simply as 'one of them.'
Kati, as Katalin Karikó prefers to be called, received the Nobel Prize for 'discoveries concerning nucleoside base modifications that enabled the development of effective mRNA vaccines against COVID-19.' The molecule at the heart of this award is pseudouridine — the same one Karikó wore around her neck as a pendant at the European Human Genetics Conference 2025, organized by the European Society of Human Genetics (ESHG), held in Milan, Italy, from May 24 to 27. On the final day of the event, Karikó delivered the Mendel Lecture, the closing session of the conference since 2006, assigned to a distinguished speaker.
Karikó's life has been extremely adventurous. A biochemist by training, she emigrated from Hungary to the US and faced many setbacks throughout her scientific career — rejected papers, denied grants — but she always persevered, eventually achieving global recognition.
Welcomed with thunderous applause, Karikó opened her address by showing attendees her Nobel medal: 'I'm showing you this while you wait to receive yours,' she said. 'I always carry it with me. Today it came in handy to get past security and enter the conference, even though I didn't have a badge!' she joked.
After delivering a lecture that all attendees described as 'inspirational,' Karikó answered questions from Univadis Italy, a Medscape Network platform, offering deeper insight into key scientific topics.
Your pioneering research on mRNA revolutionized vaccine development. In your view, what are the most promising future clinical applications of this technology beyond infectious diseases?
mRNA-based therapies are increasingly becoming a reality, and there are dozens and dozens of clinical trials focused on this new class of drugs, which could be used both to prevent and treat a variety of diseases. Consider that we already have numerous therapeutic proteins approved for use today, but they are very expensive products. So why not replace these lab-produced proteins with mRNA, giving patients the opportunity to 'build' their own medicine?
Certainly, infectious diseases still represent a major focus, accounting for about 50% of the more than 150 ongoing clinical trials involving mRNA in humans. But researchers are also turning their attention to cardiovascular diseases, genetic disorders, autoimmune conditions, and cancers.
In oncology, initial efforts focused on melanoma, but now the spectrum of possible therapies is widening — both in terms of the types of cancer being addressed and the mechanisms of action, going beyond vaccines. In some cases, however, we still lack a deep understanding of the molecular mechanisms involved, and of why the same treatment can yield very different results in seemingly identical patients. We need to keep studying to better understand and find the missing answers that will lead to effective therapies.
In recent years, interest has also grown in the biomedical potential of regulatory noncoding RNAs, such as long noncoding RNAs, microRNAs, and circular RNAs. In your opinion, what are the main challenges or opportunities in translating these molecules into effective diagnostic or therapeutic applications?
RNA research is gaining more and more ground, and there are numerous potential clinical applications for the various forms of RNA, including noncoding RNAs. These are very important, for example, in regulating gene expression.
The future holds many new developments in this area, and we are undoubtedly at a pivotal time for research, both scientific and technological, in this field.
Despite a Nobel Prize, RNA-based therapies have faced — and continue to face — skepticism and obstacles, both from the public and within the scientific community. Why, in your opinion?
Society's acceptance of a scientific discovery (in this case, a drug) is a critical point and not always easy to achieve. These are complex topics, which even highly educated people might struggle to understand. But it's certainly not impossible to convey the right messages.
Today, moreover, we are witnessing very insidious communication phenomena, also tied to social media platforms, which can spread false or misleading news that people believe. The most 'clicked' news stories often gain attention not because of the information they contain, but perhaps due to a more captivating headline — one that grabs the reader's attention or plays on emotions like fear.
Few people would read an article titled 'Broccoli Is Good for Your Health,' but many more would read one titled 'Broccoli Is a Danger to Your Health.' And even if the story claiming broccoli is harmful isn't true, it will spread, and many will believe it.
What we need to do as scientists is step out into the open, continue to communicate information accurately, and use language that is understandable even to those outside the field. That's also why we're here today and doing this interview: It's essential that there's a joint effort to ensure that scientific progress reaches everyone — and reaches them in the right way — by combating the many fake news stories and communication failures in today's world.
Many general practitioners (GPs) were on the front lines of mRNA vaccine communication and delivery. In your view, are they prepared to clearly explain how this platform works and its safety?
GPs are undoubtedly very important in the chain of communication with patients. Education and information must keep up with medicine's new opportunities, even though it's not always easy for a physician to integrate this knowledge into their daily practice.
Certainly, the relationship of trust that a GP builds with their patients has been, and still is, a key factor in explaining how these vaccines work and what the benefits and risks of new mRNA-based drugs are.
Let's not forget that the pandemic brought vaccines — and RNA more broadly — into the spotlight, but other therapies based on this nucleic acid were already circulating and will likely become increasingly common in the future.
What can your experience teach us about the long-term value of basic research, even when it initially seems to lack direct practical applications?
It shows us that investing in basic research is worthwhile, even if a project may seem extremely far from practical application to a stakeholder. Science advances in small steps, often thanks to coincidences or fortuitous meetings, but discoveries that emerge from basic research can provide answers to extremely important practical questions for humanity.
At one point in your career, you were forced to leave your university position because you 'didn't bring in enough money.' Yet not even that setback convinced you to give up research. Where did you find the motivation?
'If you really want to do something, you'll find a way; otherwise, you'll find an excuse.' I'm convinced of that, and I like to repeat it.
To researchers and to everyone with a project, I say: Don't waste time on complaints or negative thoughts. Don't let others tell you who you are or define you. Be yourself, believe in yourself, and focus on what comes next. And always find the time and willingness to be kind to the people you meet — and to say thank you. These are simple gestures, but they make a difference.
This article was translated from Univadis Italy.