Immunotherapy for brain tumors carries special risks, study finds

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.”