Scientists from the University of California, San Francisco, have developed a new cancer treatment that completely removed one of the most deadly types of childhood brain cancer in lab mice. Perhaps even more impressive, the cancer did not return when scientists tried to introduce it again.
According to ScienceDaily, the experimental treatment completely eradicated diffuse midline glioma (DMG) tumors in all mice tested across two models.
DMG is an aggressive type of brain cancer that mostly impacts children and young adults. It usually grows in the brainstem, which controls functions like breathing, heartbeat and swallowing. The illness is very hard to treat because the tumors grow into brain tissue. Nearly all children diagnosed with DMG die within a year.
New Cancer Treatment Activates Only After Entering The Brain
The experimental treatment, named B-SYNC CAR-T, uses modified immune cells to fight cancer.
CAR-T treatment is not new. Doctors have already used it successfully against some blood cancers. However, using it against brain tumors has been much more difficult. Scientists at UCSF believe they have found a way to address that challenge.
Their treatment teaches cells to stay inactive until they find a specific protein in the brain. Once activated, the cells create receptors that bind to proteins on cancer cells and attack them.
The goal of the study was to target tumors without activating those cancer-fighting receptors elsewhere in the body.
“It’s been a struggle to make CAR-T cells that overcome DMG without causing unacceptable side effects,” UCSF neurosurgery professor Hideho Okada said. “By limiting B-SYNC’s CAR expression to when and where it matters — in the brain — we saw consistent, total shutdown of DMG tumors in the lab.”
Scientists initially delivered the engineered cells through the blood. Later they tried injecting them directly into the brain’s fluid-filled ventricles. That second approach produced the remarkable results.
Scientists Hope To Begin Human Trials In 2027
The scientists also looked into whether the treatment could stop cancer from returning, SciTechDaily reported.
Seven weeks after the removal of the original tumors, they injected the treated mice with new cancer cells. Every mouse rejected the cancer cells. In addition, the modified immune cells continued to work weeks after treatment.
“We didn’t expect B-SYNC CAR-T cells to necessarily persist,” researcher Senthilnath Lakshmanachetty said. “But they are still patrolling for cancer over 70 days out — and they’re still very functional.”
Scientists are now preparing for tests at UCSF Health, possibly starting as early as April 2027. Of course, there is a big difference between removing cancer in lab mice and treating people. The therapy has not yet been proven safe or effective for humans.
Still, for a disease that so often has dire outcomes and few treatment options, these results give researchers a new and hopeful path.