Disease-fighting Promise Of MRNA

In recent years, mRNA technology enabled the rapid development of vaccines to fight COVID-19, saving millions of lives.

That same mRNA-powered approach to medicine—in which synthetic mRNA is introduced into our cells and triggers an immune response—has enormous potential to produce treatments for a wide range of human illnesses and ailments, including cancer. Most recently, mRNA—short for messenger RNA—has shown tremendous promise in the the treatment of pancreatic cancer in a phase 1 clinical trial.

"We're in a technological revolution in cancer research," says Elizabeth Jaffee, deputy director of the Sidney Kimmel Comprehensive Cancer Center. " ... If we can push this vaccine method forward, we will definitely have an impact in cancer that's going to be unexpected but almost miraculous over the next five to 10 years.

But potential cuts to federal funding for mRNA research and clinical trials threaten that progress.

Last week, three Johns Hopkins experts—Jaffee, Jeff Coller, Bloomberg Distinguished Professor of RNA Biology and Therapeutics, and Jordan Green, professor of biomedical engineering—participated in a virtual briefing to discuss the science behind mRNA, its wide-ranging applications, and how cuts to federal funding imperil U.S. scientific leadership on mRNA technologies.

How do mRNA medicines work?

Coller: An mRNA medicine is a synthetic mRNA, a piece of genetic information, that we introduce into the cell in combination with a lipid nanoparticle that goes into the cytoplasm and then makes a protein of our design.

Green: [mRNA medicine] enables genetic surgery: Being able to precisely heal the body at the molecular, genetic source of the disease, rather than the conventional approach of just treating the symptoms, which is how many medicines right now work. ... In many ways, it's like designing a rocket ship to Mars, ... but instead of going out to outer space, it's going to inner space—his fantastic voyage through our bodies to go to the right cells, to deliver the right instructions, to then create a whole paradigm of healing in the body.

What are some uses for mRNA medicine?

Coller: Many people think that mRNA only came on the scene in the year 2020, but it has a 65-year history. mRNAs were first discovered in the 1960s, and their use as medicine was explored heavily in the early 2000s. ...

mRNA became a household word in the year 2020 with the advent of the COVID-19 pandemic. We all remember what happened during this time. It was really the leadership of President Trump that led to Project Operation Warp Speed ... where we began to harness the power of mRNA to fight this pandemic.

In addition to using mRNAs to treat infectious diseases like COVID and other viruses, mRNAs are now being explored to do other intervention, including the treatment of cancer, metabolic and genetic disorders, cardiovascular diseases, and other rare genetic disorders.

Jaffee: 80% of cancers just for some reason don't alert the immune system to the fact that they are growing and progressing. Well, then comes mRNA vaccines. ... These agents have the ability to activate, even in patients with cancer, an immune response. ...

Due to the investments at the NIH [into] the new technologies, we're in a technological revolution in cancer research. ... If we can push this vaccine method forward, we will definitely have an impact in cancer that's going to be unexpected but almost miraculous over the next five to 10 years.

Image caption: Jordan Green (second from right) pictured in his lab with three fellow researchers

Image credit: Will Kirk / Johns Hopkins University

Green: mRNA delivery is a general tool for a new paradigm of medicine. So it's much broader than vaccines that can be used in a preventative way; it can be used therapeutically in many ways as well and really touches just about every area of medicine and human health. It can enable very precise and controllable expression of desired genes, and it's the expression of genes that ultimately really lead to all of our different diseases, so this is a technology that can then be utilized to address virtually all human diseases. ...

For example, type 1 diabetes. This is caused by the body's immune system attacking its own insulin-producing beta cells in the pancreas. To treat the disease symptoms, a patient has to frequently inject themselves with insulin and make up for the insulin not being produced. With mRNA technology, we could treat this at the source by reprogramming the immune system to protect and not harm the insulin producing beta cells. This would essentially cure the disease. This would be wonderful for the patients and their families, and would also significantly reduce health care costs. Lifelong diseases that require constant drug dosing could instead become a one-and-done cure.

Are mRNA medicines safe?

Coller: The science is really clear. mRNA medicines are extremely safe. There are publications that have studied well over 100 million vaccinated individuals that were treated with the COVID vaccines, and the number of adverse events that are seen is similar to what is seen with other vaccines. And in addition, it's been shown that the project Operation Warp Speed saved well over 20 million lives. ...

What's really important from a medical standpoint is that mRNA is ... destroyed over time in a very programmed manner. It dissipates, and this information then is lost. That becomes critical because it's cleared from the body and becomes very safe for the use in a medical standpoint.

What do cuts to research funding mean for mRNA medicine, including the cancer clinical trials currently being done at Hopkins?

Jaffee: We are very concerned and our patients are concerned. We don't have a lot of funding for doing these trials beyond the National Institutes of Health. The way things are going, I have to be honest, you're told a day in advance that your grant is cut, so it will be an acute change for patients who are currently being treated in our clinical trials.

Coller: I'm one of the founders of an organization called the Alliance for mRNA Medicines, and this organization has done a number of studies on the impact of potential federal cuts to mRNA research on this industry. ... In March of 2025, we conducted a survey—and this is important to note that this survey was done before the changes at the FDA—where over 100 [leading] life science professionals responded about how they felt federal cuts would influence the mRNA space. And the results of that were really quite clear. Over half of all of the leaders that we surveyed said that these cuts would delay initiatives in the mRNA space and that they would have to seek alternative funding sources and may indeed have to terminate specific programs. And perhaps most devastating is that about 30% of them would consider relocating their operations to other countries if the United States pulled away from mRNA technologies. ... Over half of all of our survey participants reported that they are already experiencing a direct impact of the policy changes that have come forth from the administration in the last few months. ...

[mRNA medicine will] represent an over $30 billion industry by the year 2030, and so it's not only a biomedical revolution, it's an economic opportunity for the United States. ... About half of all mRNA companies are housed within the United States, but by [2030], we expect that the rest of the world will really catch up. If we reduce our investment into the mRNA space, then these other countries will definitely dominate and America will lose its leadership. And with the threat of indiscriminate cuts to mRNA programs, governments around the world, like China and the European Union, Australia and Brazil, are actually not retreating from this technology. They're doubling down. Efforts to undermine mRNA technology, as we're seeing by recent actions at the NIH and FDA, will force these potential cures, therapies, and related manufacturing jobs to go overseas.