Experimental mRNA Vaccine Hints at Potential Against Glioblastoma #sciencefather #Researchawards#Brai..

 

Although immunotherapy is now a mainstay treatment for many cancers, it has yet to break through as a treatment for brain cancer, including the most common type, glioblastoma.

But recent results from several small clinical trials have hinted at the possibility of reversing this trend. Among them are findings from a very small trial testing a cancer treatment vaccine developed by researchers at the University of Florida.

Like two of the approved COVID vaccines and some other experimental cancer vaccines, this one uses genetic material called mRNA that is loaded into fat-coated nanoparticles. But it also has a unique design aspect compared with these other vaccines: the nanoparticles have layers of fat inside, making it possible to layer each one with large numbers of mRNA.

In a study published May 1 in Cell, the researchers reported that the vaccine was effective in pet dogs with naturally occurring brain cancers, allowing them to live much longer than would typically be expected.

They also reported some initial findings from a clinical trial involving four people with glioblastoma. Because it was the vaccine’s first time being used in humans, the trial’s primary goal was to ensure that the treatment was feasible and, secondarily, to look for evidence that it helps the immune system recognize and potentially attack tumors.

The trial largely succeeded on both fronts. The vaccine appeared to kick-start a robust immune response in all four participants, with rapid and dramatic changes in the levels and activity of immune system components needed to eliminate tumors.

Not unexpectedly, all four participants also had side effects from the treatment, explained the study’s lead investigator, Elias Sayour, M.D., Ph.D., a pediatric oncologist at the University of Florida and a member of the university’s McKnight Brain Institute. But although some side effects were serious, in all cases they could be successfully managed, Dr. Sayour said.

Several immunotherapy experts stressed that there’s still a long way to go before it’s known whether the vaccine is in fact safe and, as importantly, can help people live longer. 

Dr. Sayour agreed. Although these initial findings are encouraging, “we’ve only treated four patients with a few [vaccine] doses,” he warned. “There are still a lot of unknowns to work through.”

They hope to address many of these questions in additional small clinical trials of the vaccine already in the works, he said, including one in children with brain cancers. 

Alerting the immune system that something dangerous is happening 

One of the most lethal types of brain cancer, glioblastoma is largely resistant to nearly all treatments researchers have thrown at it, including targeted therapies and, more recently, immunotherapies.

While interest in mRNA vaccines has skyrocketed in the wake of the COVID pandemic, studies of mRNA cancer treatment vaccines have been underway for more than a decade. When it came to developing an mRNA vaccine to test against glioblastoma, Dr. Sayour and his team felt that the standard approach wasn’t going to cut it. 

“We felt like we needed [to try] new approaches that really shock the [immune] system,” he said.

Although the mRNA vaccines for COVID are remarkably effective, there’s a big gulf between launching an immune response against a virus trying to gain a foothold in the body and against large, established tumors tucked deep in the brain.

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