Glioblastoma is one of the deadliest brain cancers. It grows quickly, resists treatment and leaves patients and families facing a devastating truth: even the best available standard of care is rarely enough to stop it.

WashU Medicine physician-scientists refused to accept those odds. Their research led to the development of a personalized vaccine designed to help the immune system recognize and attack each patient’s tumor.

A phase 1 trial involved patients with an unmethylated MGMT subtype of glioblastoma, an especially aggressive form of the cancer. Conducted at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine, the trial found that the vaccine caused no serious side effects and prolonged patients’ overall survival compared with historical outcomes after surgery and chemo-radiotherapy.

The study was led by Tanner M. Johanns, MD, PhD, an assistant professor in the Division of Oncology who treats patients at Siteman and is a research member there.

“This kind of vaccine is a first for glioblastoma,” said Johanns.

In the trial, two-thirds of the patients had no progression six months after surgery, and two-thirds survived one year. Historically, only about 40% of glioblastoma patients reach either benchmark. One-third of participants were still alive after two years, including one patient who remains alive and recurrence-free nearly five years after diagnosis — an extraordinary milestone.

Because glioblastoma is especially difficult for the immune system to recognize and stop, the vaccine is designed around what makes each patient’s tumor unique. Using an algorithm developed at WashU Medicine, researchers identified distinguishing proteins found on a patient’s cancer cells, then created a DNA-based vaccine to help the immune system recognize those telltale proteins and attack the tumor.

The next step for researchers is to assess the vaccine’s efficacy in a larger group of patients and expand treatment to all types of glioblastomas.

The work reflects what scientific research is built to do: move bold ideas from the lab toward the people who can’t wait a second longer.