Glioblastoma is one of the deadliest forms of brain cancer, and despite surgery, radiation, and chemotherapy, the disease almost always returns. Part of the reason is that these tumors are remarkably good at evading the immune system.
Now, researchers say a personalized vaccine may offer a new way forward by teaching the immune system to recognize multiple targets on a patient’s tumor at once. The early-stage clinical trial, co-led by researchers at Washington University School of Medicine in St. Louis, found that the vaccine was safe and triggered strong immune responses that appeared to help some patients remain cancer-free for longer after surgery.
The findings, published in Nature Cancer, come from a study led jointly by Mass General Brigham and Geneos Therapeutics, a biotechnology company based in Philadelphia.
“We are extremely encouraged by these results,” said study lead author Tanner Johanns, an assistant professor in the Division of Oncology at WashU Medicine, in a press release. “This kind of vaccine is a first for glioblastoma, and it is exciting to think how we can leverage this individualized therapeutic DNA cancer vaccine platform to make a positive impact on the lives of patients who are fighting this disease.”
Creating a Unique Vaccine for Every Cancer Patient
Glioblastoma affects roughly four in 100,000 people in the U.S, and is considered the most aggressive form of brain cancer. Researchers describe it as a “cold” tumor, meaning it is especially good at hiding from the immune system.
According to Johanns, previous immunotherapies for glioblastoma have struggled to stop tumors from returning because the cancer can evolve quickly and escape immune attacks.
To address this, researchers developed a treatment using engineered DNA molecules designed to activate the immune system against each patient’s specific cancer. Because every tumor contains unique proteins, the vaccine was customized to help each patient’s immune system recognize and attack their own cancer cells.
During the trial, two-thirds of patients showed no signs of cancer progression six months after surgery, and two-thirds survived at least one year. According to the press statement, only around 40 percent of glioblastoma patients reach either milestone.
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Vaccine Can Adapt to Tumor Changes in Real Time
After surgeons removed as much of the tumor as possible, researchers analyzed the cancer tissue and designed a vaccine tailored specifically to that patient. Rather than using a universal treatment, each vaccine was personalized to the tumor’s unique genetic profile.
Importantly, the vaccine was designed to target multiple cancer markers simultaneously. That means even if the tumor changes and loses some of those markers, the immune system may still be able to recognize others.
“We chose a DNA-based platform because it would allow us an opportunity to target more cancer proteins than any vaccine had targeted before,” said Johanns.
Researchers said the platform could train the immune system to recognize as many as 40 cancer proteins unique to each patient’s tumor, about twice as many targets as previous cancer vaccines had targeted.
Early Positive Results Leave Researchers and Patients Hopeful
The study involved just nine adult patients recently diagnosed with glioblastoma and was a Phase 1 trial, meaning its primary purpose was to evaluate safety. Researchers caution that the findings do not mean there is a cure, and much larger studies will be needed to confirm the results.
Still, because glioblastoma has seen few major treatment advances in decades, even modest improvements in survival are considered meaningful.
“We are fortunate and blessed to be at the right place and at the right time, to be part of this clinical trial and have a small part in the battle against this terrible disease,” said Scott Garland, whose wife participated in the study, in the statement.
A third of participants were still alive after two years, roughly double the historical survival rate for this type of aggressive glioblastoma. One patient even remains alive and cancer-free nearly five years after her diagnosis.
“Combination therapies leveraging this personalized platform are currently being investigated at WashU to test if outcomes may be improved further,” added Johanns.
This article is not offering medical advice and should be used for informational purposes only.
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Article Sources
Our writers at Discovermagazine.com use peer-reviewed studies and high-quality sources for our articles, and our editors review for scientific accuracy and editorial standards. Review the sources used below for this article:
- This article references information from a study published in Nature Cancer: Adjuvant personalized multivalent neoantigen DNA vaccination for MGMT unmethylated glioblastoma: a phase 1 trial













