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UCSF Scientists Eliminate Deadly Brain Tumors in Mice with B-SYNC Therapy

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UCSF Scientists Eliminate Deadly Brain Tumors in Mice with B-SYNC Therapy

SAN FRANCISCO, November 10, 2026 — Scientists at the University of California, San Francisco have developed an engineered immune cell therapy that completely eliminated a deadly childhood brain cancer in mice. The treatment, called B-SYNC CAR-T, wiped out diffuse midline glioma in every animal tested across two models and showed signs of lasting protection.

How the Two-Step Activation Works

The therapy uses a molecular switch designed to keep the engineered T cells inactive while they circulate in the blood and spleen. A sensor on the cells detects brevican, a protein widely distributed throughout the brain. Only after sensing brevican do the cells produce chimeric antigen receptors that target two tumor proteins, EphA2 and IL13Rα2. This design aims to restrict the cancer-fighting activity to the brain, potentially limiting side effects elsewhere in the body.

Results Across Two Mouse Models

In experiments published October 28 in Science Translational Medicine, researchers implanted mice with human DMG cells. When B-SYNC CAR-T cells were delivered directly into the brain’s fluid-filled ventricles, tumors were eliminated in every treated mouse. Conventional CAR-T cells failed to control the cancer in the same experiments. The modified immune cells remained functional for more than 70 days after treatment. When researchers reintroduced cancer cells nearly seven weeks later, the treated mice rejected the new tumors.

Comparison With Conventional CAR-T Approaches

CAR-T therapy has transformed treatment for certain blood cancers, but brain tumors have remained difficult to target. An earlier UCSF therapy called E-SYNC, developed by the same team, used a sensor for EGFRvIII, a marker found in only about 20 percent of glioblastoma patients. By switching the activation trigger to brevican, which is present throughout the brain, B-SYNC is designed to work regardless of specific tumor mutations. Hideho Okada, MD, PhD, professor of Neurological Surgery at UCSF and co-corresponding author of the study, said limiting CAR expression to the brain produced consistent, total shutdown of DMG tumors in the lab.

Path Toward Human Trials

Diffuse midline glioma grows into the brainstem, a region that controls breathing, heartbeat, and swallowing. Nearly all children diagnosed with the disease die within a year. Researchers are now preparing for a clinical trial at UCSF Health that could begin as early as April 2027. The proposed treatment would use each patient’s own T cells, genetically modified for the therapy. Dr. Sabine Mueller, director of the UCSF Pediatric Brain Tumor Center, who will lead the first clinical trial, called the preclinical data very promising. The encouraging part is that mice with DMG are being cured in these models. Patients should talk to their doctor about what these early findings suggest for future treatment options.

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Photo: TECNIC Bioprocess Solutions / Unsplash (Unsplash License)

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