Seattle Children’s has opened a pediatric trial of intra-CNS chimeric antigen receptor (CAR) T-cell therapy for relapsed or refractory HER2-positive CNS tumors.

In this new phase 1 trial, known as BrainChild-01, CAR T cells are reprogrammed to target the protein HER2, which is expressed by many common pediatric brain tumors, including medulloblastoma, ependymoma and glioma. (DIPG is excluded from BrainChild-01 but will be included in future trials.)

By targeting HER2, the CAR T cells find and destroy cancerous tumor cells while preserving healthy brain tissue, which does not express HER2.

CNS tumors are the leading cause of cancer-related death in children under the age of 19. BrainChild-01 provides a novel therapy for children and young adults with CNS tumors who have not responded to frontline therapy and would otherwise have limited treatment options.

BrainChild-01 bypasses the blood-brain barrier.

CAR T cells are infused through an indwelling catheter, either into the resection cavity or the CNS ventricular system, depending on the tumor’s location.

Lead investigator Dr. Nick Vitanza believes introducing CAR T cells directly into the brain will be more effective than IV blood infusion since the T cells will not need to penetrate the blood-brain barrier. He also hopes patients may have fewer side effects, like neurotoxicity and cytokine release syndrome, because the reprogrammed T cells will not circulate widely through the blood.

BrainChild-01 uses methods developed by Dr. Michael Jensen and the team at the Ben Towne Center for Childhood Cancer Research. It is the next step in Seattle Children’s quest to harness the immune system and bring better therapies and cures to children worldwide. With one of the most robust pipelines of open T-cell immunotherapy clinical trials for children and young adults, Seattle Children’s is working to improve this therapy for a variety of childhood cancers to the point that it helps patients achieve long-term remission — and ultimately — a cure.

For more information or to refer a patient, contact us.

Other open immunotherapy trials:

  • PLAT-02: Phase 2 trial of a CD19-specific CAR T-cell product; open to patients with CD19-positive recurrent/refractory ALL (ages 1 to 26).
  • PLAT-03: Phase 1 study of administering CD19t transgene-expressing, T-cell antigen-presenting cells after CD19-CAR T cells, with the goal of increasing durability of remission by prolonging the persistence of CD19-CAR T cells.
  • PLAT-04: Phase 1 trial of a CD22-specific CAR T-cell product for CD22-expressing ALLs, in particular, for CD19-negative ALL relapsing after CD19-CAR T-cell immunotherapy. Open to patients with CD22+ recurrent/refractory ALL (ages 1 to 26).
  • PLAT-05: Phase 1 trial of an autologous T-cell product that co-expresses two CARs (anti-CD19 and anti-CD22) for bispecific targeting of ALL as a strategy to decrease post-remission relapses that occur by selection of antigen escape leukemic variants.
  • ENCIT-01: Phase 1 trial testing CAR T-cell therapy in children and adolescents with recurrent or refractory neuroblastoma.

To refer a patient or for more information, email us at immunotherapy@seattlechildrens.org.

Learn more about Seattle Children’s Brain Tumor Program or our Cancer and Blood Disorders Center.