Researchers from the University of Florida have developed a new mRNA cancer vaccine to retrain the body’s immune system to attack and potentially treat glioblastoma, the most common type of brain cancer affecting about 3 in every 100,000 people globally each year. Glioblastoma is challenging to treat, with an average five-year survival rate of 6.9%. The new vaccine aims to enhance responses for difficult-to-treat brain cancers and was designed based on the same technology used to create mRNA vaccines for the coronavirus. By targeting the unique mRNA repertoire of a patient’s cancer, the vaccine can be personalized to individual tumors in a feasible and commercializable manner.
For the study, researchers tested the vaccine on 10 pet dogs with natural brain tumors that had no other treatment options, observing that the dogs treated with the mRNA cancer vaccine lived an average of 139 days compared to the expected 30- to 60-day survival rate. The successful results on animals led to a small FDA-approved clinical trial involving four human study participants with glioblastoma. The researchers reported that within 48 hours of receiving the vaccine, they were able to see brain tumors transitioning from a “cold” silenced immune response to a “hot” active immune response, indicating a rapid activation of the immune system against cancer. While it is still early to assess the clinical effects of the vaccine, human study participants either lived disease-free for longer than expected or survived longer than expected, prompting the researchers to consider a larger cohort and launch the vaccine against pediatric brain tumors.
Dr. Wael Harb, a hematologist and medical oncologist not involved in the study, expressed excitement about the innovative approach using the systemic immune response to reprogram the tumor microenvironment for glioblastoma treatment. He highlighted the need for safety and efficacy data from a larger and more diverse patient population to understand how the RNA-LPAs work in reprogramming the tumor microenvironment. Dr. Jose Carrillo, a neurologist and neuro-oncologist not involved in the study, also acknowledged the promising technique for inducing an immune response in glioblastoma and expressed cautious optimism towards the progress being made in discovering new treatments for patients. Overall, the researchers believe that the new mRNA cancer vaccine could provide a head start to the immune system in the fight against rapidly evolving tumors and hope that this approach leads to better outcomes for patients with glioblastoma and potentially other cancers in the future.
While further research is needed to validate the findings and understand the safety and efficacy of the mRNA cancer vaccine in a larger patient population, the initial results from both animal and human studies show promising potential for reprogramming the immune system to fight cancer. By educating the immune cells on how to target specific cancer cells, the vaccine offers a personalized and innovative approach to glioblastoma treatment. The researchers aim to advance to phase II trials and expand the platform to pediatric brain tumors, emphasizing the importance of biomarkers to monitor the effects of the vaccine and translate the results from animal studies to human clinical trials. Through ongoing research and clinical trials, scientists and healthcare professionals are working towards developing new and effective treatments for glioblastoma and other challenging cancers, bringing hope to patients and caregivers in the fight against this deadly disease.