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The success of CAR-T immunotherapies against blood cancers has raised hope that CAR-T can bring breakthroughs to brain cancers, which haven’t had new treatments for decades. The initial clinical trials have yielded progress against brain tumors and new, potentially fatal, risks.

Three years ago, Michelle Monje, a pediatric neuro-oncologist at Stanford University, and her colleagues began a trial testing a CAR-T therapy for diffuse midline glioma, a brain cancer that primarily affects pediatric and young adult patients and occurs in places like the brain stem and thalamus. “These are nearly universally fatal cancers,” Monje said. “When we started the trial, we saw responses right away, more than we’d seen in any other context. What was amazing as a neurologist and oncologist was to see that kids got better.”


But that only came after a period of danger. Immunotherapy, including CAR-T, can cause inflammation and swelling around the tumor site. That’s fine in most of the body, but untreated inflammation from immunotherapy in the brain can lead to serious neurological symptoms, damage to vital structures, or death. That presents a new, unique toxicity to immunotherapy for nervous system tumors that Monje and her colleagues described in Nature Medicine on Thursday.

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