Researchers from the University of Florida and the University of Texas MD Anderson Cancer Center have made significant strides in developing mRNA-based treatments aimed at activating the body's immune defenses against cancer. This advancement is the culmination of over a decade of research and is considered a major milestone in the quest for a universal cancer vaccine that could enhance the effects of immunotherapy.
The analysis, which scrutinized medical records from more than 1,000 patients at MD Anderson, is still in its preliminary stages. However, if forthcoming randomized clinical trials validate these findings, the implications for cancer care could be transformative. Senior researcher Elias Sayour, M.D., Ph.D., a pediatric oncologist at UF Health and the Stop Children's Cancer/Bonnie R. Freeman Professor for Pediatric Oncology Research, emphasized the potential for this research to revolutionize oncologic care. "We could design an even better nonspecific vaccine to mobilize and reset the immune response, essentially creating a universal, off-the-shelf cancer vaccine for all cancer patients," he stated.
Dr. Jeff Coller, a leading mRNA expert at Johns Hopkins University, remarked on the implications of these findings, noting that they showcase another way that Operation Warp Speed—the U.S. government's rapid COVID-19 vaccine initiative—continues to positively influence medical advancements. "The results from this study demonstrate how powerful mRNA medicines are and how they are revolutionizing our treatment of cancer," Coller commented.
Presented on October 19 at the 2025 European Society for Medical Oncology Congress in Berlin, this study builds upon eight years of research led by Sayour, focusing on the integration of lipid nanoparticles with mRNA. Messenger RNA, or mRNA, is a crucial component present in every cell, carrying instructions for protein synthesis. In July, Sayour's team made an unexpected discovery: a robust immune response against cancer could be triggered without targeting a specific tumor protein. Instead, they found that simply stimulating the immune system to act as if combating a viral infection was sufficient.
By pairing their experimental nonspecific mRNA vaccine with immune checkpoint inhibitors—common cancer drugs that assist the immune system in recognizing and destroying tumors—the researchers observed a significant antitumor response in mice. This experimental vaccine, while not specific to COVID-19 or any other virus or cancer, utilized similar technology to that of COVID-19 vaccines. This breakthrough prompted former UF researcher and current MD Anderson scientist Adam Grippin, M.D., Ph.D., to explore whether the COVID-19 mRNA vaccine could similarly enhance immune responses in cancer patients.
The research team analyzed data from patients diagnosed with Stage 3 and 4 non-small cell lung cancer and metastatic melanoma who received treatment at MD Anderson between 2019 and 2023. Their findings revealed that patients who received a COVID mRNA vaccine within 100 days of commencing immunotherapy exhibited significantly longer survival rates compared to those who did not receive the vaccine. According to Sayour, the most notable improvements were observed in patients who, based on tumor biology and other factors, were not expected to respond well to immunotherapy.
While these results stem from an observational study and require validation through randomized clinical trials, researchers stress their potential significance. Duane Mitchell, M.D., Ph.D., Grippin's doctoral mentor and director of the UF Clinical and Translational Science Institute, underscored the urgency of conducting confirmatory research. "This is the type of treatment benefit we strive for and hope to see with therapeutic interventions," he said, "but we rarely do."
The study included records from 180 advanced lung cancer patients who received a COVID vaccine within a 100-day period before or after beginning immunotherapy and compared them with 704 patients treated with the same drugs who did not receive the vaccine. The results indicated a nearly doubled median survival rate—from 20.6 months to 37.3 months—among those who received the vaccine. In the case of metastatic melanoma patients, the median survival increased from 26.7 months to a range of 30 to 40 months, with some patients still alive at the time of data collection, suggesting that the vaccine's effects might be even more pronounced.
Researchers observed that receiving non-mRNA pneumonia or flu vaccines did not lead to any changes in longevity. To validate their findings, UF researchers utilized mouse models to combine immunotherapy drugs with a targeted mRNA vaccine aimed at the COVID spike protein. These experiments demonstrated that the combination could transform unresponsive cancers into responsive ones, effectively inhibiting tumor growth. "When you give an mRNA vaccine, it acts as a flare that mobilizes immune cells from unfavorable areas like the tumor to favorable areas like the lymph nodes," Sayour explained.
The next phase involves launching a large clinical trial through the UF-led OneFlorida+ Clinical Research Network, a consortium of hospitals and health centers across several states. Betsy Shenkman, Ph.D., who leads the consortium, emphasized the motivation to translate academic discoveries into real-world applications. If confirmed, the findings could pave the way for an even better nonspecific universal vaccine, potentially offering advanced cancer patients invaluable benefits in terms of extended survival and improved quality of life. "If this can double what we're achieving currently, or even achieve incremental improvements, that means a lot to those patients," Sayour stated.
This groundbreaking study was funded by the National Cancer Institute and various foundations. Researchers Sayour, Grippin, and Mitchell hold patents related to UF-developed mRNA vaccines, which are licensed by iOncologi Inc., a biotech company that originated as a spinout from the University of Florida, in which Mitchell has a vested interest.
