AANS Neurosurgeon | Volume 27, Number 3, 2018

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Seattle Children's Opens Immunotherapy Trial For Children With Relapsed Central Nervous System Tumors That Delivers CAR T Cells Directly Into the Brain

CAR T-cell immunotherapy targeting HER2 protein on cancer cells aims to provide children and young adults who have limited treatment options with a second line of defense

 

Seattle Children’s has opened a pioneering chimeric antigen receptor (CAR) T-cell immunotherapy trial for children and young adults with relapsed or refractory HER2-positive central nervous system (CNS) tumors where CAR T cells will be delivered directly into the brain. In the phase I trial, BrainChild-01, cancer-fighting CAR T cells will be infused through a catheter, either into the cavity where the tumor has been removed or the CNS ventricular system, depending on the location of the tumor.

CNS tumors, which are found in the brain and spine, are the most common form of childhood cancer and are the leading cause of cancer-related death in children under the age of 19. More than 4,000 children in the U.S. are diagnosed with brain tumors each year. While approximately 70% of children with newly diagnosed CNS tumors survive with standard therapy, the disease is often fatal for the approximately 30% of children who relapse.

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