A promising human clinical trial conducted on four patients could pave the way for a new treatment for glioblastoma, an aggressive form of brain cancer, writesEuronews.
Researchers at the University of Florida, USA, have developed an mRNA cancer vaccine that triggers the immune system to target the tumor.
Statistics show that around 19,000 people in the EU are affected by this condition every year.
The approach to treating glioblastoma has seen little evolution since the early 2000s, relying mainly on chemotherapy, radiation therapy, and surgery. The average survival time for patients diagnosed with this condition is about 15 months.
The team published their findings in the journal Cell earlier this month. The vaccine uses the immune system to fight cancers that are difficult to treat.
It uses a version of mRNA technology similar to that used in the COVID-19 vaccines but with a few modifications.
First, the vaccine uses cells from the patient’s tumor to create a personalized treatment.
In addition, it includes a newly developed delivery system to generate a rapid immune response, which involves injecting not individual particles but “groups of particles that wrap around each other,” said lead author Elias Sayour, in – a statement.
“And the reason we did this in the context of cancer is that these clusters alert the immune system in a much more profound way than individual particles would,” he added.
Turning the immune system against the tumor
The scientists took genetic material called RNA from the surgically removed tumor from each patient.
They then amplified messenger RNA (mRNA), which contains instructions for what’s inside every cell, including tumor cells.
This mRNA was wrapped in special lipid nanoparticles, creating a high-tech package. When these modified tumor cells were injected back into the patients’ bloodstream, they looked like viruses, triggering an immune system response.
“In less than 48 hours, we could see these tumors go from what we call ‘cold’ – immune cold, very few immune cells, very silent immune response – to ‘hot,’ very active immune response,” he said Sayour.
“This was very surprising given how quickly this happened, and what we learned is that we were able to activate the early part of the immune system against these cancers very quickly, and this is critical to unlocking the effects further effects of the immune response”.
The study is the result of encouraging findings following seven years of research that began with experiments in preclinical mouse models and progressed to a clinical trial involving ten pet dogs with advanced brain cancer.
Dogs can also develop spontaneous brain tumors that result in death, the researchers said.
The ten pet dogs lived an average of 139 days, while the average survival for dogs with the condition is 30-60 days.
Professor Duane Mitchell, co-author of the paper, said these results were “a really important finding because we often don’t know how well preclinical studies in animals will translate into similar responses in patients.”
“And while vaccines and mRNA therapies are certainly a hot topic since the COVID pandemic, this is a new and unique way to deliver mRNA to generate these really significant and rapid immune responses that we’re seeing in animals and to people”.
A phase 1 clinical trial will now test the vaccine in 24 adult and pediatric patients to confirm the results.
More research is needed to find the best way to trigger the immune system while limiting potential side effects.
