The Promise of mRNA Technology
Vaccines utilizing messenger ribonucleic acid (mRNA) technology have been in the news often the past few years, but mRNA vaccines have been under development since the 1990’s. The first clinical trial using ex-vivo human cells began in 2001. The first in-human vaccine trial was initiated in 2008. And the first use of mRNA vaccine targeting an infectious agent began in 2013. As a result, when the Covid-19 pandemic rapidly spread on a global scale there was already decades of science that allowed for the expedient development and scale-up of mRNA vaccine candidates, which ultimately saved millions of lives. As noted in one scientific journal, "[t]he truly remarkable pace of this scientific effort is unprecedented in the history of vaccines and medicine in general."
This astounding success of mRNA development and applications during the pandemic has spurred scientists to accelerate their efforts in investigating the use of mRNA technology to treat a myriad of medical conditions. Researchers have made significant strides in developing mRNA-based vaccines to combat avian flu and other viruses and to treat genetic disorders, autoimmune conditions, and even cancer.
Despite promising results thus far, the scientific community still faces significant hurdles – both medical and sociological - to the design and delivery of safe, efficacious, and trusted mRNA-based treatments.
This article will explore the development and potential of mRNA technology, its application in cancer treatment, the ongoing efforts to create a bird flu vaccine, and the challenges faced by the scientific community.
What is mRNA Technology?
Messenger ribonucleic acid (mRNA) occurs naturally in the human body. As its name implies, mRNA's function is to carry instructions to the parts of our cells that make proteins. Our bodies then use those proteins to function properly, such as growing muscles or producing antibodies to fight off infections.
Unlike traditional vaccines that use weakened or inactivated viruses, mRNA vaccines (like the COVID-19 vaccines developed by Pfizer/BioNTech and Moderna) use a small piece of the virus’ own mRNA to "teach" cells how to make a protein that triggers an immune response. The mRNA vaccines are not gene therapy. They do not alter DNA or even enter the nucleus of cells where DNA is stored. True to their nature, they act as a "messenger," delivering instructions to make the specific protein that triggers the immune response.
Encouraged by the success of the COVID-19 vaccines, scientists are now applying their newfound understanding of mRNA technology to oncology and other medical fields, but with one key difference. The mRNA vaccines for COVID-19 are prophylactic. In contrast, scientists hope to use cancer mRNA vaccines for treatment. Cancer mRNA vaccines would activate a patient's immune system to mount an attack on certain types of cancer cells. More specifically, cancer vaccines would induce strong responses from a patient's "T cells." The ultimate effect of the vaccine is to engineer a potent "cytotoxic" T cell response to locate and destroy targeted cancer cells.
mRNA Cancer Vaccines
A January 10, 2025, article in the research journal Cell Press reports more than 120 clinical trials of mRNA vaccines to treat various malignancies, including lung, breast, prostate, melanoma, and more challenging cancers such as pancreatic and brain tumors.
A number of these trials have shown dramatic results. For example, a personalized mRNA-based cancer vaccine, given in combination with an immunotherapy treatment, reduced the risk for disease recurrence or death in roughly half of patients with high-risk melanoma compared to immunotherapy treatment alone. This led the FDA to grant Breakthrough Therapy Designation to the mRNA/immunotherapy combination. Phase 3 trials are now underway. The same vaccine candidate is also being studied for possible treatment of certain types of bladder carcinoma and non-small cell lung cancer.
Another promising result is in the treatment of kidney cancer. In one study, all nine patients with advanced kidney cancer in an early-phase trial of a personalized mRNA therapeutic vaccine had successful anti-cancer immune responses and remained cancer-free approximately three years after treatment.
Many other studies utilizing mRNA technology treatments are in progress. While surgery, chemotherapy, radiation, and other cancer treatments have significantly improved patient outcomes in recent decades, the development of mRNA vaccines would be transformative. Such vaccines feature rapid design and production capabilities, high adaptability, and the potential for personalized therapeutic approaches tailored to the unique tumor profiles of individual patients. By leveraging the body's cellular machinery to produce tumor-specific antigens, mRNA vaccines can elicit a robust and targeted immune response against cancer cells.
Bird Flu Vaccine
Over the past several years, research into an mRNA vaccine for the H5N1 flu, colloquially known as bird or avian flu, has become a priority for health officials. Since the late 1990s, bird flu has been known to affect wild birds and poultry, with occasional spillover to humans. In 2021, however, a major outbreak of a strain that can infect many mammal species, most notably cattle, has spread around the globe. While no human-to-human spread has yet been detected, the Centers for Disease Control and Prevention (CDC) have reported 66 human cases and one fatality from animal-to-human infections. If mutations in the virus were to eventually allow human-to-human spread, then a vaccine would be necessary.
Traditional vaccine production methods (including recombinant, cell based, and egg based) are not likely to be replaced by mRNA technology any time soon as each platform has its benefit and challenges. Some of the benefits of mRNA technology is that there is no risk of recipient infection as there is no use of live-attenuated virus or virus vectors in the vaccine. Additionally, since the platform does not require the chemical and cell culture processes of traditional methods and therefore manufacturing complexity, scale-up and batch completion times can be reduced along with cost. However, studies show that mRNA vaccines may not be retained by the body as long and therefore immune-response protection can be reduced more rapidly than traditional methods. For these reasons, scientists are seeking to develop an mRNA vaccine while also working to enhance long term immune response. Early studies have shown promising results, and the FDA has approved two Phase 1 trials in humans. In addition, in January 2025, the U.S. Department of Health awarded Moderna a $590 million contract for mRNA-based bird flu vaccines.
The Path Forward
Groundbreaking treatments for a plethora of medical conditions based on mRNA technology are just over the horizon. Yet, much research needs to be completed to assess fully the risk of adverse effects and to improve the expression, duration, and stability of any mRNA-based vaccines developed.
Sociological hurdles are also now in play. The rapid pace with which the COVID-19 vaccine was developed depended, in large part, upon the federal government's willingness to commit significant funds for research and work in close collaboration with scientists at drug companies and in academia. However, recent shifts in government objectives include the reevaluation of federal research funds, particularly those concerning mRNA vaccines.
Life science industry participants are further concerned with sentiments regarding mRNA vaccine research expressed on both the federal and state level through laws and policy. Although no bans have yet been passed, some states such as Texas and Iowa have introduced legislation targeted specifically at restricting mRNA vaccines. Robert F. Kennedy Jr., the newly confirmed Health and Human Services (HHS) Secretary, also has expressed strong views on vaccines and mRNA technology. It remains unclear what his leadership of HHS will mean exactly for continued mRNA vaccine research and development.
Modern mRNA technology represents a groundbreaking advancement in medical science, offering unprecedented speed and adaptability in vaccine development. From its remarkable success in combating COVID-19 to its promising applications in cancer treatment and avian flu prevention, mRNA technology is poised to revolutionize healthcare. However, the path forward is fraught with challenges, including scientific hurdles and sociopolitical obstacles. Continued research, funding, and public support are essential to fully harness the potential of mRNA technology and ensure its benefits reach patients worldwide. As we stand on the cusp of a new era in medicine, the promise of mRNA technology offers hope for a healthier future.
Authored by Chris Dorko, Berkley Life Sciences, VP, Risk Management Resources Officer